Articles

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
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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Business
April 2, 2019
AveXis Expands Gene Therapy Manufacturing Capacity with Acquisition
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Business
April 1, 2019
EMA Grants Krystal Biotech PRIME Designation for DEB Gene Therapy
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Business
April 1, 2019
FDA Grants AstraZenca and Merk’s NF1 Drug Breakthrough Therapy Designation
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Drug Development
April 1, 2019
FDA Orphan Drug Designations Q1 2019
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Rare Advocate
March 29, 2019
How Misperceptions Can Create Barriers to Care
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Uncategorized
March 28, 2019
Rare Leader: Mary Anne Meskis, Executive Director, Dravet Syndrome Foundation
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Business
March 28, 2019
Polyneuron Raises $22.6 Million to Advance Autoimmune Disease Therapies
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Drug Development
March 28, 2019
Orchard Therapeutics Reports MLD Gene Therapy Shows Sustained Benefit
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Awareness
March 28, 2019
Seeing Sickle Cell as a Blood Disease
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Finance
March 27, 2019
Imara Raises $63 Million, Brings in Pfizer Rare Disease Head as CMO
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Deals
March 27, 2019
ProQR and EB Research Form Drug Company
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Research
March 27, 2019
NIH Gives $3.2 Million Grant to OMRF to Study Sarcoidosis
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Drug Development
March 26, 2019
FDA Grants Promedior Breakthrough Therapy Designation for Idiopathic Pulmonary Fibrosis Drug
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Drug Development
March 25, 2019
FDA Issues Draft Guidance on Natural History Studies for Rare Diseases
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Business
March 22, 2019
Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs
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Uncategorized
March 21, 2019
Rare Leader: Melissa Bryce Gamble, Executive Director, The Global Foundation for Peroxisomal Disorders
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Business
March 21, 2019
FDA Approves Jazz Pharmaceuticals’ Narcolepsy Drug
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Policy
March 21, 2019
Medicare Part D Spending on Specialty Drugs Climbs to $32.8B in 2015
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Business
March 21, 2019
FDA Grants Enzyvant Fast Track and Rare Pediatric Disease Designations for Farber Disease Therapy
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Business
Rare Advocate
March 20, 2019
Cure SMA and Parent Project Muscular Dystrophy Form Collaboration to Collect RWD
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Business
March 20, 2019
Alexion Teams Up with Zealand, Affibody, in Pair of Development Deals
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Business
March 20, 2019
Pfizer Takes Stake in Gene Therapy Developer Vivet
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Business
March 20, 2019
GW Pharmaceuticals Sells Priority Review Voucher for $105 Million
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Drug Development
March 18, 2019
Gottlieb Calls on Industry to Modernize Approach to Clinical Trials
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Drug Development
March 15, 2019
Getting Misfolding Proteins to Shape Up
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Rare Advocate
March 14, 2019
Rare Leader: Ashley Valentine, Co-Founder, Sick Cells
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Rare Disease
March 13, 2019
A High School Student Unlocks A Rare Disease Mystery
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Research
March 13, 2019
NDF Awards $700,000 in Grants for GNE Myopathy Research and Programs
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Policy
March 12, 2019
Report Calls on NICE to Use Greater Flexibility in Reviewing Orphan Drugs
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Business
March 8, 2019
A Different Approach to Inhibiting the Complement System
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Rare Advocate
March 7, 2019
Rare Leader: Haley Oyler, President, SETBP1 Society
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Policy
March 7, 2019
Patient Group Calls on BIO to Disavow “Price Gouging” by LEMS Drugmaker
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Business
March 6, 2019
Alnylam Reports Positive Results from Late-Stage Study of Givosiran in AHP
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Awareness
March 6, 2019
FDA to Hold Public Meeting on the Impact of Rare Diseases
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Awareness
March 6, 2019
How Maria Voermans Found Her Calling
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Drug Development
March 4, 2019
Oxford, Harrington Discovery Institute Form Center to Advance Rare Disease Therapies
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Business
March 4, 2019
Biogen to Acquire Nightstar Therapeutics for $800 Million
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Business
March 2, 2019
Perlara to Wind Down Operations
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Awareness
March 1, 2019
Looking Back in Time to Find Rare Disease Patients Today
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Awareness
February 27, 2019
A Rare Disease Gets Rock Star Treatment
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Business
February 27, 2019
Amicus Establishes Global Research and Gene Therapy Center of Excellence
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Deals
February 25, 2019
Roche Agrees to Acquire Spark Therapeutics for $4.8 Billion
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Business
February 25, 2019
Ipsen to Acquire Clementia Pharmaceuticals for up to $1.3 Billion
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Business
February 22, 2019
Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company
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Policy
February 21, 2019
Catalyst Pharmaceuticals Defends Price of LEMS Drug in Letter to Senator Bernie Sanders
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Awareness
February 21, 2019
Rare Leader: Connie Lee, President and CEO, Angioma Alliance
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Business
February 21, 2019
Avexis Plans $60 Million Expansion of Manufacturing Facility in North Carolina
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Awareness
February 21, 2019
Teaching Doctors When to See If a Horse Has Stripes
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Diagnosis
February 20, 2019
Global Commission Releases Plan for Shortening Rare Disease Diagnostic Odyssey
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Business
February 19, 2019
Nestle Health Sciences Exercises Option for Codexis PKU Treatment
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Business
February 15, 2019
Passage Bio Raises $115.5 Million to Develop Gene Therapies for CNS Disease
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Business
February 15, 2019
Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline
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People & Organizations
February 15, 2019
Rare Leader: Lori Sames, CEO, Hannah’s Hope Fund
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Drug Development
February 13, 2019
Study Questions the Value of Priority Review Vouchers as Incentives
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Business
February 12, 2019
Neurogene Raises $68.5 to Advance Gene Therapies for Rare Neurological Diseases
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Genomics
February 12, 2019
Combining Proteomics with Genomics Helps Pinpoint a Rare Disease
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Innovation
February 12, 2019
Rare Disease Groups Launch Newborn Screening Study in Australia
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Business
February 12, 2019
FDA Grants Clementia Rare Pediatric Disease Designation by FOP Drug
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Awareness
February 8, 2019
Connecting Rare Disease Patients in India and the United States
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Drug Development
February 8, 2019
Solid Biosciences Reports Disappointing Preliminary Results of DMD Gene Therapy Study
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Drug Development
February 8, 2019
First Results of In Vivo Gene Editing Trial of MPS II Treatment Show Only Small Benefit
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Business
February 7, 2019
FDA Approves Sanofi’s Cablivi for Rare Blood-Clotting Disorder aTTP, the First of a New Class of...
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Rare Advocate
February 7, 2019
Rare Leader: Liz Marfia-Ash, President and Founder, GRIN2B Foundation
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Awareness
Policy
February 5, 2019
Sen. Bernie Sanders Investigates $375,000 Price of LEMS Drug
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Business
February 4, 2019
Alexion and Caelum Enter $550M Collaboration in AL Amyloidosis
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Rare Disease
February 4, 2019
UK Grant Supports Diagnosis and Treatment of Rare Diseases
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Innovation
February 4, 2019
ASCO Names Progress in Treating Rare Cancers Advance of the Year
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Business
February 1, 2019
Regenerative Medicine Moves into the Spotlight
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News

December 4, 2019
Giving Back this Holiday Season
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December 4, 2019
Five Rare Disease Podcasts to Get You Through Your...
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December 2, 2019
FDA Accepts Incyte’s Cholangiocarcinoma Candidat...
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October 30, 2019
5 Tips to Celebrating a Spooktacular Rare Hallowee...
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October 15, 2019
2019 Cox Scholarship for RARE Compassion – A...
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