Articles

People & Organizations
February 27, 2020
Rare Leader, Glenna Spencer Steele, Executive Director of Glut1 Deficiency Foundation
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Drug Development
February 27, 2020
A Mother’s Journey to Becoming a Citizen Scientist
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Business
February 26, 2020
FDA Grants Fast Track Designation for Lysogene’s Gene Therapy for Sanfilippo Syndrome Type A
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Business
February 26, 2020
FDA Grants Fast Track Designation to Translate Bio’s mRNA Therapy for Cystic Fibrosis
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Legislation
February 25, 2020
Rare Disease Advocates Take to the Hill
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Business
February 25, 2020
FDA Approves Asuragen’s Genetic Test to Diagnose Fragile X Syndrome
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Business
February 25, 2020
Raremark Partners with TriNetX to Connect Rare Disease Patients to Clinical Trials
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Business
February 24, 2020
Cell and Gene Therapy Companies Raised $6.4 Billion in 2019 for Rare Disease
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Research
February 24, 2020
Study Suggests Need for a Precision Approach to Metabolic Diseases
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Business
February 24, 2020
Galderma Reports Positive Results from Mid-Stage Study of Therapy for Rare Skin Condition
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Business
February 21, 2020
Targeting a Common Link in Otherwise Different Rare Diseases
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Business
February 21, 2020
FDA Grants BioMarin Priority Review for Hemophilia A Gene Therapy
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Business
February 21, 2020
The Lost Art of Letter Writing
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People & Organizations
February 20, 2020
Rare Leader, Jennifer Canvasser, Founder, NEC Society
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Business
February 20, 2020
Spruce Biosciences Raises $88 Million to Advance Rare Endocrine Disease Pipeline
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Business
February 20, 2020
FDA Grants Forma Rare Pediatric Disease Designation for SCD Therapy
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Business
February 20, 2020
EC Approves Pfizer’s Vyndaqel for ATTR-CM
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Business
February 19, 2020
CANbridge Raises $98 Million to Advance Rare Disease Pipeline
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Business
February 19, 2020
Univercells Scores Up to $54 Million Investment from KKR
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Business
February 19, 2020
Avrobio, Catalyst, Complete Offerings
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Business
February 18, 2020
Gene Therapy Biotech Adverum Raises $150 Million in Public Offering
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Healthcare
February 18, 2020
First UK Patients Begin Gene Therapy Treatment for Blindness through NHS
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Drug Development
February 18, 2020
ALS Association Grants Neuropore $500,000 to Explore Treatments that Inhibit Neuronal Damage
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Drug Development
February 14, 2020
Bringing Clinical Trials to Patients
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Research
February 14, 2020
Gene Therapy and Gene Editing Combo Shows Potential for Treating Some Metabolic Disorders
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Research
February 14, 2020
Researchers Find Evidence of Childhood Rare Disease in 60 Million-Year-Old Dinosaur Tumor
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People & Organizations
February 13, 2020
Rare Leader: Susan Foley, Executive Directors, HESA
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Business
February 13, 2020
Synspira and CF Foundation Collaborate to Develop Enzyme Replacement for Malabsorption Syndromes
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People & Organizations
February 12, 2020
Bucking Up Students with Rare Disease
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Business
February 12, 2020
Ligand Buys Icagen’s Core Assets, Partnered Programs with Roche and CF Foundation
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Research
February 11, 2020
Patient-Partnered Research Points to Genetic Cause and Potential Treatment for Rare Cancer
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People & Organizations
February 11, 2020
Uplifting Athletes and Horizon Team up for NFL Scouting Combine Reps for Rare
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Research
February 11, 2020
Cures Within Reach Seeking Repurposing Proposals for LMIC Countries
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Business
February 10, 2020
Lilly and Genentech Therapies Fail in Trial for Rare Form of Alzheimer’s Disease
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Drug Development
February 10, 2020
Study Finds Treatment for Rare Eye Disease May Prevent Blindness
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Drug Development
February 7, 2020
Crowdsourcing Therapeutic Approaches for an Ultra-Rare Disease
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Business
February 7, 2020
Zogenix Reports Positive Results from Pivotal Trial of LGS Therapy, but Shares Drop
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People & Organizations
February 6, 2020
Rare Leader: Harsha Rajasimha, CEO, IndoUSrare
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Uncategorized
February 6, 2020
Chiesi Establishes Global Rare Diseases Division
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Business
February 6, 2020
Azafaros Raises $28 Million to Advance Rare Metabolic Disorders Pipeline
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Business
February 6, 2020
Beam Therapeutics Raises $180 Million in IPO
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Rare Disease
February 5, 2020
A Call for Precise Rare Disease Definitions
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In Rare Form
February 5, 2020
Finding Missing Pieces to Genetic Puzzles
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Regulatory
February 5, 2020
FDA Releases Guidance for Sanfilippo Syndrome
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Drug Development
February 4, 2020
Chan Zuckerberg Initiative Awards $13.5 Million to Rare Disease Patient-Led Groups
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Business
February 3, 2020
Mammoth Raises $45 Million For CRISPR Therapeutics and Diagnostics
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Business
February 3, 2020
GW Pharmaceuticals Applies for FDA Approval for Epidiolex to Treat TSC
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Business
February 3, 2020
AgeX Teams with UC Irvine on Stem Cell Research for Neurological Disorders
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Business
January 31, 2020
Delivering Oligonucleotide Therapies to Any Cell Type
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Patient Tools
January 31, 2020
New Report Shows Key Policy Improvements in 2019 but More Progress Needed to Help People with...
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People & Organizations
January 30, 2020
Rare Leader: Marianne Clancy, Executive Director, Cure HHT
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Drug Development
January 30, 2020
Sanofi Reports Positive Results in Study of Experimental Niemann-Pick Therapy
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Regulatory
January 30, 2020
FDA Grants Aprea Breakthrough Therapy Designation for MDS Therapy
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Regulatory
January 30, 2020
FDA Grants ProQR Rare Pediatric Disease Designation Rare Eye Disease Therapy
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Business
January 29, 2020
A Building Boom for Gene Therapy Facilities
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Business
January 29, 2020
Rare Skin Disease Drug Developer Timber Agrees to Reverse Merger with BioPharmX
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Business
January 29, 2020
Akcea Reports Positive Results from Hypertriglyceridemia Therapy Study
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Rare Patient
January 28, 2020
Rare Disease Patients More Likely to Suffer Depression and Anxiety
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Business
January 28, 2020
FTC, N.Y. AG Sue Vyera Pharmaceuticals, Shkreli over Alleged Anticompetitive Scheme
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Regulatory
January 28, 2020
FDA Issues Regulatory Guidance to Provide Clarity on Gene Therapy Development
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Business
January 28, 2020
Quench Bio Raises $50 Million to Advance Therapies for Inflammatory Diseases
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Business
January 28, 2020
Passage Bio Names Bruce Goldsmith as CEO
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Drug Development
January 28, 2020
Acceleron PAH Therapy Meets Endpoints in Mid-Stage Trial
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Business
January 28, 2020
Ipsen Pauses Dosing in Study of Drug It Acquired in $1.3B Acquisition of Clementia
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Drug Development
January 24, 2020
Putting Scientific Standards Under the Microscope
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Business
January 24, 2020
Protalix Looks Beyond Biobetters
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Business
January 24, 2020
FDA Approves First Treatment for Rare Soft Tissue Cancer
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People & Organizations
January 23, 2020
Rare Leader: Eric Cole, Executive Director, Dandy-Walker Alliance
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Business
January 23, 2020
Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SMA Type 1 Trial in Infants
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Business
January 22, 2020
Making the Value Proposition for Rare Disease Therapies
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Business
January 22, 2020
FDA Approves First Treatment for Rare Thyroid Eye Disease
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Business
January 22, 2020
Quest Diagnostics Expands Rare Disease Offerings with Acquisition of Blueprint Genetics
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Business
January 21, 2020
Stealth Initiates Clinical Study of Second-Generation Mitochondrial Candidate in Neurodegenerative Diseases
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Policy
January 20, 2020
FDA to Move to Online Submissions for Orphan Drug Designation Requests
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Drug Development
January 17, 2020
Driving N-of-1 Therapies for Ultra-Rare Disease Patients
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Drug Development
January 17, 2020
Creating the Means to an End
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People & Organizations
January 16, 2020
Rare Leader: Daniel Lewi, Founder and CEO, Cure & Action for Tay-Sachs Foundation
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Uncategorized
January 16, 2020
FDA Clears GeneTx and Ultragenyx to Begin Angelman Syndrome Trial
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Business
January 16, 2020
Cyprium Receives Rare Pediatric Disease Designation for Menkes Therapy
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Business
January 16, 2020
Raremark Expands Leadership Team
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Business
January 14, 2020
Ionis Enters into Discovery and Development Deals with Empirico, Aro
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Business
January 14, 2020
Nationwide Children’s Hospital Launches Affiliate to Manufacture Gene Therapies
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Business
January 14, 2020
EUSA Pharma Enters Collaboration with BeiGene for Two Rare Disease Therapies
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Business
January 13, 2020
Generation Bio Completes $100 Million Financing to Advance Re-Dosable Gene Therapy Platform
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Deals
January 13, 2020
LogicBio Enters Collaboration with Takeda to Develop New Genome Editing Therapy
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Business
January 13, 2020
Ultragenyx Reports Positive Topline Results from OTC Deficiency Gene Therapy Study
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Business
January 10, 2020
Turning Good Thoughts into Good Actions
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Awareness
January 10, 2020
Rare Leader: Emily Kramer-Golinkoff, Co-Founder, Emily’s Entourage
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Business
January 10, 2020
Cutting the Time and Cost of Drug Discovery with MicroOrgans
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Business
January 10, 2020
FDA Approves First Targeted Therapy to Treat Rare Mutation in Patients with GIST
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Business
January 10, 2020
FDA Grants Aruvant Rare Pediatric Disease Designation for SCD Therapy
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Business
January 9, 2020
Translational Medicine Accelerator and Cyclica Form Rare Disease Accelerator
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Business
January 9, 2020
Catabasis and Duchenne UK Partner to Evaluate Experimental DMD Drug
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Business
Business
January 9, 2020
Regeneron Reports Positive Data in Mid-Stage Ultra-Rare Bone Disease Drug Study
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Drug Development
January 8, 2020
Ionis Founder Launches Nonprofit to Produce N-of-1 Therapies
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Business
January 8, 2020
VectivBio Launches with $35 Million to Develop Medicines for Serious Rare Diseases
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Business
January 8, 2020
Apellis’ Pegcetacoplan Bests Soliris in Phase 3 PNH Study
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Diagnosis
January 7, 2020
Senators Introduce Bill to Expand Access for Kids to WGS Through Medicaid
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Business
January 7, 2020
Deep Genomics Raises $40 Million in Series B Financing
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Business
January 7, 2020
Saniona Names SOBI’s Rami Levin as CEO
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Business
January 7, 2020
Rhythm CEO to Step Down After FDA Filing
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Diagnosis
January 6, 2020
NHS England Using DNA Testing to Accelerate Diagnosis for Critically Ill Kids
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Business
January 6, 2020
FDA Grants QED Fast Track and Orphan Drug Designations for Rare Cancer Therapy
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Business
January 3, 2020
Acquisition Combines Two Pipelines for Rare Skin Diseases
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Pricing
January 3, 2020
NICE Rejects Akcea’s FCS Drug Waylivra as Too Costly
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Uncategorized
January 2, 2020
Rare Leader: Christina SanInocencio, Executive Director, LGS Foundation
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Business
January 2, 2020
Savara Wins Breakthrough Therapy Designation for Rare Lung Disease Therapy
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Business
December 31, 2019
A Down Year for Drug Approvals, But Notable for Its Standouts
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Business
December 31, 2019
Complete FDA Orphan Drug Approvals in 2019
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Business
December 31, 2019
FDA Orphan Drug Designations in Q4 2019
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Business
December 30, 2019
Fulcrum Enters Drug Discovery Collaboration with Acceleron for Undisclosed Pulmonary Disease
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Drug Development
December 27, 2019
Programming Cells to Produce Neuroprotective Factors to Treat ALS
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Diagnosis
December 26, 2019
Rare Leader, David Jacob, Founder and CEO of ThinkGenetic
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In Rare Form
December 24, 2019
The Making of a Neuroscientist
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Business
December 23, 2019
Sarepta In Blockbuster Deal with Roche for Ex-U.S. Rights to its Experimental DMD Gene Therapy
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Business
December 20, 2019
Origin’s Rapid Path to Seek Approval for Treatment of Ultra-Rare Metabolic Condition
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Business
December 20, 2019
Stealth BioTherapeutics PMM Therapy Fails in Late-Stage Trial
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Business
December 20, 2019
Duke University Establishes Translational Research Collaboration with $130 Million from Deerfield
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Business
December 20, 2019
Ultragenyx Sells Future Royalties on Crysvita for $320 Million
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People & Organizations
December 19, 2019
Rare Leader: Charlene Son Rigby, President, STXBP1 Foundation
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Business
December 18, 2019
Epirium Raises $85 Million to Develop Treatments for Rare Neuromuscular Diseases
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Business
December 18, 2019
Global Blood Therapeutics and Syros Pharmaceuticals in Rare Blood Disorders Pact
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Business
December 18, 2019
Chondrial Merges with Zafgen, Advances FA Candidate
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Business
December 18, 2019
Eiger Initiates Rolling Submission Progeria Drug for FDA Approval
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Drug Development
December 18, 2019
Nurturing a Culture for Translational Science
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Business
December 17, 2019
Triplet Therapeutics Launches with $59 Million to Develop Treatments for Repeat Expansion Disorders
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Uncategorized
December 17, 2019
Amylyx Reports Results from ALS Trial
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Business
December 17, 2019
Wave Discontinues Development of Experimental DMD Drug
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Uncategorized
December 17, 2019
Alnylam Reports Positive Results from Late-Stage PH1 Study of Lumasiran
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Business
December 17, 2019
Proteostasis Reports Positive Study Results from Combination Therapy for CF
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Business
December 16, 2019
BioMarin Reports Positive Final Results from Late-Stage Study of Achondroplasia Drug
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Business
December 16, 2019
Biogen Reports It Will Discontinue Development of Drug for Progressive Supranuclear Palsy
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Business
December 16, 2019
CHMP Issues Positive Opinion for Pfizer’s ATTR-CM Drug
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Business
December 16, 2019
FDA Grants Mirum Rare Pediatric Disease Designation for Maralixibat in Alagille Syndrome
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RARECast Podcasts
December 13, 2019
Developing a Common Language for Rare Diseases
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Business
December 13, 2019
FDA Grants Accelerated Approval to First Targeted Treatment for Rare DMD Mutation
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Diagnosis
December 12, 2019
Perkin Elmer Wins FDA Authorization for First Newborn Test for DMD
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Awareness
December 12, 2019
Rare Leader: Justin Porcano, Co-Founder and Executive Director, Save Sight Now
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In Rare Form
December 11, 2019
The Other Data Drug Developers Need to Consider
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Business
December 10, 2019
Merck to Acquire ArQule for $2.7 Billion
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Business
December 10, 2019
Quellis Biosciences Raises $17 Million to Develop Rare Disease Therapies
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Awareness
December 10, 2019
Invitae and BioMarin Launch Genetic Testing Program for Skeletal Dysplasias
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Business
December 10, 2019
Sanofi Presents Positive Results from Pivotal Trial of Sutimlimab in people with CAD
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Business
December 10, 2019
ASH Meeting Offers Updates on Several Rare Disease Therapies in Development
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Business
December 9, 2019
Jasper Therapeutics Complete $35 Million Financing to Expand Curative Therapies
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Business
December 9, 2019
Healx and Boehringer Ingelheim Partner in Rare Neurological Diseases Pact
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Innovation
December 9, 2019
Andra Stratton, Co-Counder of Lipodystrophy United, Discusses the Future of Rare Disease
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Business
December 6, 2019
Limelight Bio Raises $75 Million to Develop Next-Gen Gene Therapies
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Business
December 6, 2019
FDA Rejects Enzyvant’s Rare Disease Therapy over Manufacturing Issues
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Drug Development
December 6, 2019
Why Payers Lag Drug Developers and Regulators in Embracing Real-World Evidence
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Drug Development
People & Organizations
December 5, 2019
Rare Leader: Stacie Lindsey, Founder, CEO, Cholangiocarcinoma Foundation
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Business
December 5, 2019
Regeneron Reports Positive Data with Pozelimab in Patients with a Rare Blood Disorder
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Business
December 4, 2019
Censa Reports Positive Results in Phase 2 Trial in Patients with PKU
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Business
December 3, 2019
Astellas Moves into Gene Therapy with $3 Billion Acquisition of Audentes
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Business
December 2, 2019
Neurocrine and Xenon Partner to Develop Treatment for Rare Epilepsy
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Innovation
December 2, 2019
Harsha Rajasimha, Co-founder of ORDI in USA, Discusses the Future of Rare Disease
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Awareness
November 29, 2019
How a Brother’s Love Is Forging a Path for Customized Therapies for Rare Disease Patients
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Awareness
November 29, 2019
Rare Leader: Katherine Wilemon, Founder and CEO, FH Foundation
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Rare Disease
November 27, 2019
Brigham and Women’s Lauches Rare Liver Disease Center with $15 Million Gift
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Business
November 27, 2019
La Jolla Pharmaceuticals Halts Development of Beta Thalassemia Drug as CEO Departs
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Business
November 27, 2019
ChemoCentryx Reports Positive Data from Pivotal Trial of Avacopan in ANCA-Associated Vasculitis
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In Rare Form
Business
November 26, 2019
FDA Approves Novel Treatment to Target Abnormality in Sickle Cell Disease
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Business
November 25, 2019
Alexion’s Soliris Wins Japanese Approval to Treat NMOSD
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Business
November 25, 2019
FDA Grants PTC Priority Review for SMA Therapy
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Innovation
November 25, 2019
NEXT: Lara Bloom, CEO of The Ehlers Danlos Society, Discusses the Future of Rare Disease
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Policy
November 22, 2019
Addressing the Barriers to Accessing Experimental Therapies
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In Rare Form
Business
November 21, 2019
Healx Launches Rare Treatment Accelerator
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Business
November 21, 2019
CRISPR Therapeutics Initiates $275 Million Public Offering
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Business
November 21, 2019
FDA Grants Newron Rare Pediatric Disease Designation for Sarizotan in Rett Syndrome
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People & Organizations
November 21, 2019
Rare Leader: Rich Horgan, Founder and President, Cure Rare Disease
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Business
November 21, 2019
FDA Approves Alnylam’s RNAi Drug Givlaari for AHP Attacks
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Business
November 20, 2019
Reata Raises $505 Million to Advance Rare Disease Drugs
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Business
November 19, 2019
Dicerna and Novo Nordisk Partner in RNAi Therapies for Liver Diseases
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Business
November 19, 2019
Gene Editing Treatment Shows Positive Results in First Two Treated Patients with Blood Disorders
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Patient Tools
November 19, 2019
Backpack Health and Project Alive Launch Hunter Syndrome Study
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Research
November 19, 2019
Rare Genetic Variants Linked to Sudden Cardiac Death
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Business
November 19, 2019
FDA Approves Novartis Drug as First Targeted Therapy for SCD-Related Pain Crises
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Policy
November 19, 2019
Australia Commits $37.5 Million to Develop Treatments for Rare Disorders
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Business
November 18, 2019
Roche Beefs Up Its Fibrosis Pipeline in $1.4 Billion Deal to Acquire Promedior
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Drug Development
November 18, 2019
FDA Grants Anavex Rare Pediatric Disease Designation
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Drug Development
November 18, 2019
EMA Grants Orchard Accelerated Assessment of Therapy for Patients with MLD
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Innovation
November 18, 2019
NEXT: Nicole Boice, Global Genes, Discusses the Future of Rare Disease
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Rare Advocate
Deals
November 15, 2019
Sarepta and StrideBio Collaborate to Advance Novel Gene Therapies
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Diagnosis
November 15, 2019
mRNA Sequencing Ends 25-Year Diagnostic Odyssey
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Drug Development
November 15, 2019
FDA Accepts New Drug Submission for Selumetinib in NF1, grants Priority Review
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People & Organizations
November 14, 2019
Rare Leader: Kim Tuminello, Co-Founder, Assocation for Creatine Deficiencies
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Drug Development
November 14, 2019
GSK Reports Nucala Reduces Flares in Patients with HES
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Drug Development
November 14, 2019
Palvella Begins Phase 3 Portion of Pivotal Study in Pachyonychia Congenita
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Deals
November 14, 2019
Centogene and Pfizer Collaborate for Rare Disease Drug Development
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Awareness
November 13, 2019
Sharing Stories, Sharing Hope
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Finance
November 13, 2019
Avidity Raises $100 Million to Advance Treatments for Rare Muscle Disorders
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Deals
November 13, 2019
Merck Acquires Calporta for Up to $576 Million
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Innovation
November 13, 2019
FDA Grants X4 Pharma Breakthrough Therapy Designation for WHIM Therapy
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Drug Development
November 13, 2019
FDA Halts Solid Biosciences’ DMD Gene Therapy Trial
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Drug Development
November 12, 2019
Reata Reports Positive Results from Pivotal Study of Alport Syndrome Therapy
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Drug Development
November 12, 2019
Alnylam Initiates Pivotal Study of RNAi Therapeutic for PH1; Reports Positive Extension Study Results
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Innovation
November 11, 2019
NEXT: Maria Picone, TREND Community, Discusses the Future of Rare Disease
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Innovation
November 11, 2019
FDA Approves Celgene’s Therapy for Beta Thalassemia
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Drug Development
November 11, 2019
Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SMA Trial
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Policy
November 10, 2019
NICE Recommends GW Pharmaceuticals’ Epidyolex for Rare Epileptic Seizures
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People & Organizations
November 8, 2019
Turning Chronic Illness into a Fashion Statement
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People & Organizations
November 7, 2019
Rare Leader: Torie Robinson, CEO, Epilepsy Sparks
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Drug Development
November 7, 2019
Mirum Reports Positive Data from Long-Term Study in Alagille Syndrome
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Finance
November 7, 2019
Centogene Raises $56 Million in IPO for Rare Disease Diagnostics
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Finance
November 7, 2019
Arkuda Therapeutics Raises $44 Million to Treat Rare Form of Dementia
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Drug Development
November 7, 2019
Constellation Presents Positive Early Data in Myelofibrosis Study
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Research
November 6, 2019
NIH Awards New York State’s IBR $1.95 Million to Study Rare Diseases
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Diagnosis
November 6, 2019
Genomenon Releases Updated Search Engine to Accelerate Rare and Genetic Disease Diagnosis
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Patient Stories
November 4, 2019
Learning that Together, We Can Make a Difference
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Innovation
November 4, 2019
NEXT: Luke Rosen, KIF1A.org, Discusses the Future of Rare Disease
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Strategy
November 3, 2019
ARM Reports Europe Less Competitive in Attracting Advanced Therapy Clinical Trials
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Rare Advocate
November 1, 2019
Connecting Young Adults with Rare and Chronic Conditions
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Deals
October 31, 2019
Beam Therapeutics Teams with Prime Medicine for Prime Editing Technology
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People & Organizations
October 31, 2019
Rare Leader: Stephanie Gebel, CEO, The Snow Foundation
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Healthcare
October 31, 2019
Where Cutting-Edge Science Meets Horse-and-Buggy Culture
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Regulatory
October 31, 2019
FDA Places Partial Hold on Trials of Zolgensma Injected into Spinal Fluid
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Business
October 31, 2019
Catalyst Firdapse Fails in Congenital Myasthenic Syndromes Trial
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Deals
October 30, 2019
Vertex Exercises Cystic Fibrosis Gene Editing Options from CRISPR Therapeutics
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Drug Development
October 30, 2019
FDA, EMA Accept Genentech’s Application for Approval of NMOSD Therapy
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Research
October 30, 2019
Study Shows Many Common Genetic Factors Determine Severity of Rare Genetic Diseases
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People & Organizations
October 30, 2019
Cystic Fibrosis Foundation Launches $500 Million Path to a Cure Initiative
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Rare Disease
October 29, 2019
Study Estimates More Than 300 Million with Rare Disease Worldwide, but Number Likely Higher
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Innovation
October 29, 2019
NEXT: Stephen Kingsmore, Rady Children’s Institute for Genomic Medicine, Discusses the Future of Rare Disease
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Research
October 29, 2019
Researchers Discover Monkey Model for Rare Blinding Disease
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Business
October 28, 2019
FDA Grants Mirum Breakthrough Therapy Designation for Alagille Syndrome Drug
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Business
October 25, 2019
Cabaletta Bio Raises $75 Million in IPO for T-Cell Therapies for Autoimmune Diseases
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Research
October 25, 2019
MC10 and University of Vermont Form Partnership to Study Gait Abnormalities in Movement Disorders
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Rare Caregiver
October 25, 2019
Helping Caregivers Care for Themselves
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Drug Development
October 24, 2019
NIH Launches Global Collaboration to Tackle SCD, HIV with Gene Therapies
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People & Organizations
October 24, 2019
Rare Leader: Patricia Weltin, CEO, Beyond the Diagnosis
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Business
October 24, 2019
New Biotech Allievex Licenses BioMarin’s Sanfilippo ERT
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Uncategorized
October 24, 2019
Orchard Says Interim Data Suggest Frozen Gene Therapy Similar to Fresh Formulation in MLD
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Business
October 23, 2019
Moderna Receives FDA Fast Track Designation for Propionic Acidemia Program
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Uncategorized
October 22, 2019
Rethinking the Costs of Rare Disease
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Business
October 22, 2019
FDA Approves Vertex’s Triple Combo Cystic Fibrosis Treatment
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Business
October 22, 2019
Protalix Reports Positive Interim Data in Late-Stage Fabry Study
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People & Organizations
October 21, 2019
Gas Station Network Fuels Rare Disease Organization with $65,000 Donation
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Business
October 21, 2019
ProQR, Rhythm Complete Public Offerings
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Business
October 21, 2019
Alexion Receives FDA Approval for Ultomiris for aHUS
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Innovation
October 20, 2019
NEXT: James Valentine of Hyman,Phelps & McNamara Discusses the Future of Rare Disease
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Awareness
October 18, 2019
A Festival of Moving Pictures
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Business
October 17, 2019
Stealth Says Experimental Drug Improves Cardiac Function in Barth Syndrome Patients
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People & Organizations
October 17, 2019
Rare Leader: Ginger Spitzer, Past Executive Director, Foundation for Sarcoidosis Research
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Business
October 16, 2019
AI Drug Developer Healx Secures $56 million to Launch Global Rare Disease Accelerator Program
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Business
October 16, 2019
Editas and AskBio Team Up to Develop Novel Treatments for Neurological Diseases
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Business
October 16, 2019
Ipsen Expands Its Rare Disease Portfolio with License for Blueprint Medicines’ FOP Treatment
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Business
October 16, 2019
Alexion Adds to Rare Disease Pipeline with Acquisition of Achillion
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Business
October 16, 2019
FDA Grants ProQR Rare Pediatric Disease Designation for Rare Eye Disease Therapy
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People & Organizations
October 16, 2019
Global Genes Names Cox Scholarship Awardees
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Business
October 15, 2019
With Rights Back in Hand, Reata Reports Positive Results for FA Therapy
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Drug Development
October 14, 2019
The Mother of Invention Strikes Again
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Innovation
October 14, 2019
NEXT: Bruce Bloom of Healx Discusses the Future of Rare Disease
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Business
October 14, 2019
Roche Said Study Shows Its Therapy Is Superior to Commonly Used Treatment in Rare Autoimmune Disease
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Business
October 11, 2019
Deep Genomics Boasts an AI First in Drug Discovery
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Diagnosis
October 11, 2019
New Genomics Method Can Reveal the Causes of Rare Genetic Diseases
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Diagnosis
October 11, 2019
Jumping Genes Found to Cause Rare Developmental Disorders in Children
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Business
October 10, 2019
Alexion Pays Stealth $30 Million for Option to Co-Develop Late-Stage Program in Mitochondrial Diseases
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Business
October 10, 2019
UCB to Acquire Ra Pharmaceuticals in Deal Valued at $2.1 Billion
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Business
October 10, 2019
Reata Pays $330 Million to Reacquire Drug Rights from AbbVie
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Rare Advocate
October 10, 2019
Rare Leader: Keisha Greaves, Founder, Girls Chronically Rock
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Innovation
October 9, 2019
When Did Super Become a Bad Thing?
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Business
October 9, 2019
Bluebird Bio and Novo Nordisk Team Up to Develop Genome Editing Therapies
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Business
October 8, 2019
FDA Approves First Treatment to Increase Pain-Free Light Exposure in Rare Disorder
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Business
October 8, 2019
Akcea Grants Pfizer Exclusive License to Antisense Therapy
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Regulatory
October 8, 2019
FDA Awards 12 Grants to Fund Rare Disease Therapy Clinical Trials
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Policy
October 8, 2019
FDA Provides $4.1 Million for Two Natural History Studies
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Business
October 8, 2019
Avrobio Doses First Patient in Gene Therapy Trial for Cystinosis
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Business
October 7, 2019
Zogenix Presents Positive Results for Experimental Treatment for TK2 Deficiency
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Business
October 7, 2019
Audentes Therapeutics Reports Positive Data from XLMTM Gene Therapy Trial
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Rare Advocate
October 4, 2019
How to Live with a Disability with Your Mojo Intact
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Research
October 4, 2019
NIH Awards $31 Million in Funding to Bolster Rare Diseases Research Collaborations
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Business
October 4, 2019
Sarepta Therapeutics Reports Positive Results for Limb-Girdle Gene Therapy
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Research
October 4, 2019
Researchers Identify Cause of Rare Deadly Neurological Disease
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Business
October 4, 2019
Nippon Shinyaku Files for Approval of DMD Exon-Skipping Drug
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Business
October 4, 2019
Aeovian Raises $37 Million to Advance Therapy for Rare CNS Disease
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Business
October 4, 2019
Goldfinch Enters Licensing Agreement with Takeda for Rare And Metabolic Kidney Diseases
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People & Organizations
October 3, 2019
Rare Leader: Nathan Peck, CEO, Cure VCP Disease
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Business
October 2, 2019
Biogen Reports Positive Results for Pre-symptomatic Treatment with Spinraza in SMA
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Business
October 2, 2019
FDA Grants Fast Track Designation to Amryt Pharma for EB Therapy
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Business
October 2, 2019
Two Drug Developers Targeting Rare Muscle Diseases Name New CEOs
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Data Center
October 2, 2019
U.S. Food and Drug Administration Orphan Drug Approvals in 2019
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Data Center
October 2, 2019
U.S. FDA Orphan Drug Designations Q3 2019
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Business
October 1, 2019
NewLink Genetics and Lumos Merge to Focus on Rare Disease Therapies
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Uncategorized
October 1, 2019
SOBI Acquires Rare Blood Disorder Biotech Dova Pharmaceuticals
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Business
September 30, 2019
Ocugen and CanSino Biologics Enter Gene Therapy Strategic Partnership
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Business
September 30, 2019
FDA Approves Ipsen’s Dysport to Treat Upper Limb Spasticity in Children
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Business
September 25, 2019
Amryt Raises $60 Million as It Completes Acquisition of Aegerion
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Business
September 25, 2019
FDA Grants Fast Track Designation to Aldeyra Therapeutics for PRV Therapy
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Policy
September 24, 2019
Rare Disease Patients Get Nod in UN Declaration on Universal Health Coverage
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Business
September 24, 2019
ArTara Reverse Merges with Proteon and Raises $42.5 Million
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Business
September 24, 2019
FDA Grants Fast Track Designation to Annexon for Guillain-Barré Therapy
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Business
September 23, 2019
Shake-up at Ionis-Owned Akcea Removes Top Execs
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Business
September 23, 2019
EU Gives Marketing Nod to GW Pharmaceuticals for Drug to Treat Rare Epilepsies
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Business
September 23, 2019
FDA Places Hold on Abeona Ahead of Pivotal Trial of Cell Therapy for RDEB
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Business
September 22, 2019
Bluebird Bio Reports Positive Long-Term Data from CALD Gene Therapy Study
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People & Organizations
September 19, 2019
Rare Leader: Marie Conley, Founder, The Conley Cushing’s Disease Foundation
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Business
September 19, 2019
Strongbridge Biopharma Reports Late-Stage Results for Experimental Cushing’s Therapy
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Innovation
September 18, 2019
Technology Reshaping the Rare Disease Landscape, but Non-Scientific Challenges Threaten to Impede Progress
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Rare Advocate
September 18, 2019
Fighting for Other People’s Children
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Business
September 18, 2019
Versantis Raises $16.1 Million to Develop Treatment for Liver Disease Including ACLF
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Research
September 17, 2019
Scientists Find Cause, and Potential Therapeutic Strategy, for Krabbe Disease
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Business
September 17, 2019
Acceleron Scraps FSHD Treatment After Mid-Stage Trial Disappointment
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Business
September 17, 2019
FDA Approves CTD Holdings Expanded Access Treatment Program in NPC
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Business
September 16, 2019
SpringWorks Raises $162 Million in IPO
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Business
September 16, 2019
Akcea and Ionis Present Positive Early-Stage Data for Experimental ATTR Therapy
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Rare Advocate
September 13, 2019
A Doctor’s Quest to Cure His Own Rare Disease
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Business
September 13, 2019
Castle Creek Acquires Fibrocell for Gene Therapies to Treat Rare Skin Disorders
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Business
September 13, 2019
Vertex Reaches Access Agreement with Scottish Government for CF Drugs
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People & Organizations
September 12, 2019
Rare Leader: Janice Schwartz, Executive Director, Pachyonychia Congenita Project
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Business
September 12, 2019
Pharvaris Raises $66 Million to Advance Oral Therapy for Hereditary Angioedema
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Business
September 10, 2019
Trucode Launches to Advance Next-Generation Gene Editing Platform
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Business
September 10, 2019
Passage Bio Licenses Experimental CMT Gene Therapy
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Business
September 10, 2019
Mayo Clinic Enters Partnership with Google to Drive Digital Transformation of Healthcare
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Regulatory
September 6, 2019
FDA Clarifies Accelerated Pathway for Medical Devices for Rare Disease Indications
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Business
September 6, 2019
Takeda Issues Recall for Natpara
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Business
September 6, 2019
Passage Bio Raises $110 Million to Develop Gene Therapies for CNS Diseases
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Business
September 5, 2019
BridgeBio Creates New Subsidiary to Develop Therapy for Limb-Girdle Muscular Dystrophy
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People & Organizations
September 5, 2019
Rare Leader: Akiva Zablocki, President, The Hyper IgM Foundation
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Business
September 5, 2019
First Generic Orfadin Approved
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Policy
September 4, 2019
USCIS Pulls Back Plan to Deport Undocumented Patients Getting Life-Saving Treatments
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Business
September 4, 2019
Publisher Elsevier Cuts Deal with NIH’s NCATS for Use of Rare Disease Database
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Policy
September 4, 2019
NICE Recommends Use of Gene Therapy for Rare Inherited Eye Disorder
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Global Genes News
September 4, 2019
A Letter from Kimberly Haugstad
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Business
September 4, 2019
UniQure’s Late-Stage Hemophilia B Gene Therapy Study Fully Enrolled
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Business
RARECast Podcasts
August 30, 2019
The Benefits Support Groups Offer Rare Disease Patients
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Research
August 30, 2019
Researchers Discover New Biomarker for Rare Autoimmune Disease
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Business
August 30, 2019
SpringWorks Gets Breakthrough Therapy Designation for Desmoid Tumor Drug
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Rare Advocate
August 29, 2019
Rare Leader: Victoria Jackson, CEO, Guthy-Jackson Charitable Foundation
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Business
August 29, 2019
Report of Patient Deaths Creates Questions about Vertex’s CF Drug
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Business
August 29, 2019
Alexion’s Soliris Wins European Approval to Treat Rare CNS Disease
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Policy
August 27, 2019
ARM Issues Statement of Principles on Genome Editing
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Business
August 26, 2019
Zogenix to Acquire Modis Therapeutics for up to $400 Million
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Innovation
August 26, 2019
New Gene-editing Tool Could Help Treat Genetic Disorders
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Drug Development
August 23, 2019
Targeting the Epigenetic Drivers of Cancer
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Research
August 23, 2019
Gene Linked to Rare Disorder also Plays a Key Role in Alzheimer’s
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People & Organizations
August 22, 2019
Rare Leader: Isabel Jordan, Chair, Rare Disease Foundation
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Business
August 22, 2019
Pfizer to Invest $500 Million in Gene Therapy Manufacturing Facility in North Carolina
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Business
August 22, 2019
Chinook Raises $65 Million to Advance Precision Therapies for Rare Kidney Diseases
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Business
August 22, 2019
Retrophin PKAN Therapy Fails in Late Stage Trial
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Business
August 22, 2019
Sobi Sells Priority Review Voucher to AstraZeneca for $95 Million
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Business
August 14, 2019
Renovacor Raises $11 Million to Advance Gene Therapy for Rare Cardiovascular Disease
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People & Organizations
August 8, 2019
Rare Leader: Amber Freed, Founder and CEO, SLC6A1 Connect
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Business
August 8, 2019
Bayer Bets on Cell Therapy with Buyout of BlueRock Therapeutics
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Business
August 7, 2019
Novartis CEO Says He’s Committed to Rebuilding Trust After Manipulation of SMA Gene Therapy Data
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Business
August 7, 2019
Akcea’ and Ionis’ Waylivra Meets Endpoints in Study in Patients with FPL
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Business
August 7, 2019
Rhythm Reports Positive Pivotal Results from Rare Obesity Trials
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Deals
August 7, 2019
Selecta Partners with AskBio to Develop AAV Gene Therapies That Can Be Re-Dosed
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Drug Development
August 7, 2019
Critical Path Institute and NORD Launch Rare Disease Data Platform
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Business
August 7, 2019
Glycomine Raises $33 Million
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Business
August 7, 2019
Zynerba Cannabiol Shows Promise in Small Fragile X Study
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Business
August 6, 2019
Allakos Reports Positive Results in Mid-Stage Study of Therapy for Rare GI Disorders
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Global Genes
August 6, 2019
Global Genes Names Kimberly Haugstad as New CEO
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Business
August 5, 2019
Pfizer’s Sickle Cell Treatment Fails in Late-Stage Trial
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Drug Development
August 2, 2019
Seeking to Halt and Reverse Fibrotic Diseases
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Business
August 2, 2019
Cystic Fibrosis Foundation Commits Up to $15 Million to Arcturus for mRNA Therapies
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Business
August 2, 2019
Amicus Reports Positive Interim Clinical Data for Batten Disease Gene Therapy
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Uncategorized
August 2, 2019
Ultragenyx Seeks FDA Approval for Long-Chain Fatty Acid Oxidation Disorders Therapy
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In Rare Form
August 2, 2019
It’s a Low-Down Dirty Shame
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People & Organizations
August 1, 2019
Rare Leader: Jennifer Wescoe, Executive Director, Wescoe Foundation for Pulmonary Fibrosis
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Research
July 31, 2019
ANU Gets $6.9 Million Donation to Research Rare Autoimmune Condition
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Business
July 26, 2019
Allergan and Editas Begin First Human Trial of CRISPER-Based Gene Editing Therapy
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Business
July 26, 2019
Aeglea Receives FDA Breakthrough Therapy designation for Arginase 1 Deficiency ERT
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People & Organizations
July 25, 2019
Rare Leader: Robyn De Leon, Executive Director, Connecting Families UCD Foundation
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Legislation
July 25, 2019
Bipartisan Legislation Seeks to Save Government $100 Billion in Drug Costs
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In Rare Form
July 25, 2019
Four Diseases with a Common Problem
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Business
July 25, 2019
Iveric Bio Expands Gene Therapy Portfolio for Rare Retinal Diseases
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In Rare Form
Business
July 17, 2019
FDA Grants Novartis Priority Review for Sickle Cell Therapy
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Business
July 16, 2019
Recursion Pharmaceuticals Raises $121 Million to Advance Platform and Pipeline
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Business
July 15, 2019
Recordati Acquires Rights to Novartis’ Rare Endocrine Disease Drugs
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Business
July 8, 2019
Alexion Wins European Approval for Ultomiris to Treat PNH
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Business
July 8, 2019
UniQure Says Hemophilia B Gene Therapy Raises FIX Activity up to 54 Percent of Normal
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Business
June 28, 2019
BridgeBio Raises $349 Million in IPO to Fund Rare Disease Programs
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Business
Business
June 24, 2019
FDA Approves Vertex’s Cystic Fibrosis Treatment for Children Ages 6 to 11
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Business
June 24, 2019
FDA Grants Alexion Priority Review for Ultomiris to Treat AHUS
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Genomics
June 21, 2019
What Rare Disease Patients Should Understand about Genetics
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Drug Development
June 11, 2019
FDA Grants Denali Therapeutics Rare Pediatric Disease Designation for Hunter Syndrome Therapy
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Drug Development
June 7, 2019
A Gene Hunter Becomes a Drug Hunter Too
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Business
June 7, 2019
Vertex Acquires Exonics Therapeutics, Expands Deal with CRISPR Therapeutics
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Rare Advocate
June 6, 2019
Rare Leader: Sandra Bedrosian-Sermone, ADNP Kids Research Foundation
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Business
Research
June 5, 2019
Newfound Autoimmune Syndrome Can Be Treated with Anti-Inflammatory Drugs
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Drug Development
June 4, 2019
EU Grants Conditional Approval for Bluebird Bio’s Beta-Thalassemia Gene Therapy
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Drug Development
June 4, 2019
FDA Greenlights Clinical Trial of Ra Pharmaceuticals Treatment of IMNM
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Drug Development
June 4, 2019
FDA Grants SpringWorks Fast Track Designation for Experimental NF1 Therapy
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RARECast Podcasts
May 31, 2019
The Psychological Toll Rare Diseases Can Have on Healthy Siblings
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Rare Advocate
May 30, 2019
Rare Leader: Amy Brin, Executive Director, Child Neurology Foundation
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Business
May 30, 2019
Amicus and Penn Expand Gene Therapy Collaboration
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Business
May 30, 2019
Ayala Pharmaceuticals Raises $30 Million in Novartis-Led Round
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Business
May 30, 2019
Inhibrx and Chiesi Enter Agreement for AATD Candidate Worth up to $162.5 Million
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Business
May 29, 2019
Orchard Secures Exclusive Worldwide License Agreement for MPS-I Gene Therapy
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Business
May 29, 2019
GenSight Will Seek Approval for Gene Therapy for Rare Eye Disease Strength of Sham Treatment
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Business
May 29, 2019
Oxford Biomedica Secures up to $67.5 Million in Strategic Investment
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Business
May 29, 2019
Viela Bio Enters NMSOD Collaboration with Hansoh Pharma Worth for up to $220 Million
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Business
May 28, 2019
BioMarin’s Hemophilia A Gene Therapy Hits Prespecified Criteria for Regulatory Submissions
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Business
May 24, 2019
FDA Approves Gene Therapy Zolgensma for SMA; Novartis Sets Price at $2.1 Million
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Diagnosis
May 24, 2019
Blood Test Can Provide Earlier Diagnosis of Autism Spectrum Disorder
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Rare Advocate
May 23, 2019
Rare Leader: Rob Long, Executive Director, Uplifting Athletes
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Business
May 21, 2019
Reneo Raises $50 Million to Develop Therapies for Mitochondrial Diseases
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Business
May 20, 2019
FDA Expands Use of Gattex to Include Short Bowel Syndrome
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Business
May 15, 2019
Vertex and Kymera Enter Targeted Protein Degradation Collaboration
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Business
May 14, 2019
Solid Biosciences Reports Serious Adverse Event in DMD Trial
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Business
May 14, 2019
Celgene Receives Breakthrough Therapy Designation Kaposi Sarcoma Drug
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Business
May 14, 2019
UniQure Reports Promising Interim Data from Study of Hemophilia B Gene Therapy
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Research
May 9, 2019
A Roadmap for Rare Disease Groups
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Rare Advocate
May 9, 2019
Rare Leader Profile: Mark Dant, Executive Director of The Ryan Foundation
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Policy
May 9, 2019
Minnesota Senate Passes Legislation to Create Rare Disease Advisory Council
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Business
May 8, 2019
Pfizer Pays $340 Million for Achondroplasia Therapy Developer Therachon
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Business
May 8, 2019
Vielo Bio Reports Positive Pivotal Results of Treatment for Rare CNS Disorder
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Business
May 8, 2019
Gilead Enters Strategic Collaboration with Goldfinch Bio on Kidney Diseases
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Drug Development
May 7, 2019
FDA Approves First LEMS Drug for Children and Adds New Wrinkle to Pricing Controversy
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Drug Development
May 7, 2019
GW Pharma Reports Positive Results for CBD Drug in Patients with Rare Severe Epilepsy
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Drug Development
May 7, 2019
EC Approves Akcea and Ionis’ Waylivra for Rare Lipid Disorder
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Business
May 7, 2019
European Commission Approves BioMarin’s PKU Drug Palynziq
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Rare Advocate
April 25, 2019
Rare Leader: Amanda Moore, CEO, Angelman Syndrome Foundation
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Drug Development
April 25, 2019
Global Sales of Orphan Drug Expected to Grow at Twice the Rate of Non-Orphan Drugs
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Business
April 24, 2019
Fulcrum Sees Promise in Failed Heart Drug as Treatment for FSHD
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Legislation
April 24, 2019
ACE Kids Act to Improve Coordination of Care for Medically Complex Kids
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Diagnosis
April 23, 2019
A Case for Whole Genome Sequencing
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Drug Development
April 23, 2019
Report Predicts Sharp Jump in Clinical Development Productivity for Rare Disease Therapies
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Drug Development
April 22, 2019
Novartis Investigating Patient Death in SMA Gene Therapy Trial
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Research
April 22, 2019
Study Points to Lithium as Potential Treatment for Limb Girdle Muscular Dystrophy
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Awareness
April 19, 2019
A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition
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Rare Advocate
April 18, 2019
Rare Leader: Emily Milligan, Executive Director, Barth Syndrome Foundation
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Drug Development
April 18, 2019
St. Jude Gene Therapy “Cures” Babies with “Bubble Boy” Disease
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Uncategorized
April 17, 2019
When a Cold Is a Serious Threat
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Business
April 17, 2019
FDA Grants Ultragenyx Rare Pediatric Disease Designation for Experimental Therapy
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Business
April 16, 2019
Fibrocell Teams with Castle Creek on RDEB Gene Therapy
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Business
April 16, 2019
NICE Gives Akcea Nod for Tegsedi to Treat Ultra-Rare hAATR
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Business
April 9, 2019
FDA Notifies Zogenix of Inadequacies in Dravet Syndrome Drug Application
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Business
April 9, 2019
Cyclerion Taps Shire’s Andreas Busch as Chief Innovation Officer
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Business
April 8, 2019
Alnylam and Regeneron Enter Blockbuster Collaboration
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Business
April 8, 2019
Audentes Expands Platform and Pipeline with Programs for DMD and DM
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Business
April 5, 2019
How a Patient Group Helped Drive Drug Development in Rett Syndrome
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Uncategorized
April 4, 2019
Rare Leader: Julie Raskin, Executive Director, Congenital Hyperinsulism International
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Business
April 4, 2019
ICER Calls Spinraza Too Pricey, Urges Fair Pricing of Zolgensma
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Business
April 4, 2019
FDA Grants Abeona Fast Track Designation for Sanfilippo Type B Gene Therapy
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Uncategorized
Business
April 3, 2019
Dyne Therapeutics Raises $50 Million to Develop Therapies for Muscle Diseases
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Business
April 3, 2019
EspeRare Partners with Dermelix to Develop Treatment for XLHED
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Drug Development
April 2, 2019
Sangamo Provides Positive Updates on Hemophilia A and Beta Thalassemia Gene Therapy Trials
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