Articles

Drug Development
August 16, 2019
Tackling Kidney Diseases with Orally-Delivered Enzymes
Read More
Deals
August 15, 2019
Ultragenyx Enters Partnership with GeneTx to Develop Angelman Syndrome ASO
Read More
People & Organizations
August 15, 2019
Rare Leader: Sarah Chisholm, Executive Director, BPAN Warriors
Read More
Business
August 14, 2019
Renovacor Raises $11 Million to Advance Gene Therapy for Rare Cardiovascular Disease
Read More
Business
August 13, 2019
Senate Pressure Grows on Novartis over Gene Therapy Data Manipulation
Read More
Business
August 13, 2019
Jazz Pharmaceuticals Acquires Neurological Drug Developer
Read More
Business
August 13, 2019
Pharming Acquires Late-Stage Novartis Drug to Treat Ultra-Rare Immune Disorder
Read More
Diagnosis
August 12, 2019
Study Says the Internet Can Be Valuable for People with Undiagnosed Rare Disorders
Read More
Business
August 12, 2019
ProQR Gets FDA Clearance to Start Clinical Trial to Treat Rare Eye Disease
Read More
Business
August 12, 2019
Immunic Says First Patient Enrolled in Proof-of-Concept Trial for Rare Liver Disease Therapy
Read More
Business
August 9, 2019
Giving Voice to Patients through Data
Read More
Drug Development
August 9, 2019
Sarepta Says DMD Gene Therapy Adverse Event Report Submitted Erroneously
Read More
People & Organizations
August 8, 2019
Rare Leader: Amber Freed, Founder and CEO, SLC6A1 Connect
Read More
Business
August 8, 2019
Bayer Bets on Cell Therapy with Buyout of BlueRock Therapeutics
Read More
Business
August 7, 2019
Novartis CEO Says He’s Committed to Rebuilding Trust After Manipulation of SMA Gene Therapy Data
Read More
Business
August 7, 2019
Akcea’ and Ionis’ Waylivra Meets Endpoints in Study in Patients with FPL
Read More
Business
August 7, 2019
Rhythm Reports Positive Pivotal Results from Rare Obesity Trials
Read More
Deals
August 7, 2019
Selecta Partners with AskBio to Develop AAV Gene Therapies That Can Be Re-Dosed
Read More
Drug Development
August 7, 2019
Critical Path Institute and NORD Launch Rare Disease Data Platform
Read More
Business
August 7, 2019
Glycomine Raises $33 Million
Read More
Business
August 7, 2019
Zynerba Cannabiol Shows Promise in Small Fragile X Study
Read More
Business
August 6, 2019
Allakos Reports Positive Results in Mid-Stage Study of Therapy for Rare GI Disorders
Read More
Global Genes
August 6, 2019
Global Genes Names Kimberly Haugstad as New CEO
Read More
Business
August 5, 2019
Pfizer’s Sickle Cell Treatment Fails in Late-Stage Trial
Read More
Drug Development
August 2, 2019
Seeking to Halt and Reverse Fibrotic Diseases
Read More
Business
August 2, 2019
Cystic Fibrosis Foundation Commits Up to $15 Million to Arcturus for mRNA Therapies
Read More
Business
August 2, 2019
Amicus Reports Positive Interim Clinical Data for Batten Disease Gene Therapy
Read More
Uncategorized
August 2, 2019
Ultragenyx Seeks FDA Approval for Long-Chain Fatty Acid Oxidation Disorders Therapy
Read More
In Rare Form
August 2, 2019
It’s a Low-Down Dirty Shame
Read More
People & Organizations
August 1, 2019
Rare Leader: Jennifer Wescoe, Executive Director, Wescoe Foundation for Pulmonary Fibrosis
Read More
Research
July 31, 2019
ANU Gets $6.9 Million Donation to Research Rare Autoimmune Condition
Read More
Business
July 26, 2019
Allergan and Editas Begin First Human Trial of CRISPER-Based Gene Editing Therapy
Read More
Business
July 26, 2019
Aeglea Receives FDA Breakthrough Therapy designation for Arginase 1 Deficiency ERT
Read More
People & Organizations
July 25, 2019
Rare Leader: Robyn De Leon, Executive Director, Connecting Families UCD Foundation
Read More
Legislation
July 25, 2019
Bipartisan Legislation Seeks to Save Government $100 Billion in Drug Costs
Read More
In Rare Form
July 25, 2019
Four Diseases with a Common Problem
Read More
Business
July 25, 2019
Iveric Bio Expands Gene Therapy Portfolio for Rare Retinal Diseases
Read More
In Rare Form
Business
July 17, 2019
FDA Grants Novartis Priority Review for Sickle Cell Therapy
Read More
Business
July 16, 2019
Recursion Pharmaceuticals Raises $121 Million to Advance Platform and Pipeline
Read More
Business
July 15, 2019
Recordati Acquires Rights to Novartis’ Rare Endocrine Disease Drugs
Read More
Business
July 8, 2019
Alexion Wins European Approval for Ultomiris to Treat PNH
Read More
Business
July 8, 2019
UniQure Says Hemophilia B Gene Therapy Raises FIX Activity up to 54 Percent of Normal
Read More
Business
June 28, 2019
BridgeBio Raises $349 Million in IPO to Fund Rare Disease Programs
Read More
Business
Business
June 24, 2019
FDA Approves Vertex’s Cystic Fibrosis Treatment for Children Ages 6 to 11
Read More
Business
June 24, 2019
FDA Grants Alexion Priority Review for Ultomiris to Treat AHUS
Read More
Genomics
June 21, 2019
What Rare Disease Patients Should Understand about Genetics
Read More
Drug Development
June 11, 2019
FDA Grants Denali Therapeutics Rare Pediatric Disease Designation for Hunter Syndrome Therapy
Read More
Drug Development
June 7, 2019
A Gene Hunter Becomes a Drug Hunter Too
Read More
Business
June 7, 2019
Vertex Acquires Exonics Therapeutics, Expands Deal with CRISPR Therapeutics
Read More
Rare Advocate
June 6, 2019
Rare Leader: Sandra Bedrosian-Sermone, ADNP Kids Research Foundation
Read More
Business
Research
June 5, 2019
Newfound Autoimmune Syndrome Can Be Treated with Anti-Inflammatory Drugs
Read More
Drug Development
June 4, 2019
EU Grants Conditional Approval for Bluebird Bio’s Beta-Thalassemia Gene Therapy
Read More
Drug Development
June 4, 2019
FDA Greenlights Clinical Trial of Ra Pharmaceuticals Treatment of IMNM
Read More
Drug Development
June 4, 2019
FDA Grants SpringWorks Fast Track Designation for Experimental NF1 Therapy
Read More
RARECast Podcasts
May 31, 2019
The Psychological Toll Rare Diseases Can Have on Healthy Siblings
Read More
Rare Advocate
May 30, 2019
Rare Leader: Amy Brin, Executive Director, Child Neurology Foundation
Read More
Business
May 30, 2019
Amicus and Penn Expand Gene Therapy Collaboration
Read More
Business
May 30, 2019
Ayala Pharmaceuticals Raises $30 Million in Novartis-Led Round
Read More
Business
May 30, 2019
Inhibrx and Chiesi Enter Agreement for AATD Candidate Worth up to $162.5 Million
Read More
Business
May 29, 2019
Orchard Secures Exclusive Worldwide License Agreement for MPS-I Gene Therapy
Read More
Business
May 29, 2019
GenSight Will Seek Approval for Gene Therapy for Rare Eye Disease Strength of Sham Treatment
Read More
Business
May 29, 2019
Oxford Biomedica Secures up to $67.5 Million in Strategic Investment
Read More
Business
May 29, 2019
Viela Bio Enters NMSOD Collaboration with Hansoh Pharma Worth for up to $220 Million
Read More
Business
May 28, 2019
BioMarin’s Hemophilia A Gene Therapy Hits Prespecified Criteria for Regulatory Submissions
Read More
Business
May 24, 2019
FDA Approves Gene Therapy Zolgensma for SMA; Novartis Sets Price at $2.1 Million
Read More
Diagnosis
May 24, 2019
Blood Test Can Provide Earlier Diagnosis of Autism Spectrum Disorder
Read More
Rare Advocate
May 23, 2019
Rare Leader: Rob Long, Executive Director, Uplifting Athletes
Read More
Business
May 21, 2019
Reneo Raises $50 Million to Develop Therapies for Mitochondrial Diseases
Read More
Business
May 20, 2019
FDA Expands Use of Gattex to Include Short Bowel Syndrome
Read More
Business
May 15, 2019
Vertex and Kymera Enter Targeted Protein Degradation Collaboration
Read More
Business
May 14, 2019
Solid Biosciences Reports Serious Adverse Event in DMD Trial
Read More
Business
May 14, 2019
Celgene Receives Breakthrough Therapy Designation Kaposi Sarcoma Drug
Read More
Business
May 14, 2019
UniQure Reports Promising Interim Data from Study of Hemophilia B Gene Therapy
Read More
Research
May 9, 2019
A Roadmap for Rare Disease Groups
Read More
Rare Advocate
May 9, 2019
Rare Leader Profile: Mark Dant, Executive Director of The Ryan Foundation
Read More
Policy
May 9, 2019
Minnesota Senate Passes Legislation to Create Rare Disease Advisory Council
Read More
Business
May 8, 2019
Pfizer Pays $340 Million for Achondroplasia Therapy Developer Therachon
Read More
Business
May 8, 2019
Vielo Bio Reports Positive Pivotal Results of Treatment for Rare CNS Disorder
Read More
Business
May 8, 2019
Gilead Enters Strategic Collaboration with Goldfinch Bio on Kidney Diseases
Read More
Drug Development
May 7, 2019
FDA Approves First LEMS Drug for Children and Adds New Wrinkle to Pricing Controversy
Read More
Drug Development
May 7, 2019
GW Pharma Reports Positive Results for CBD Drug in Patients with Rare Severe Epilepsy
Read More
Drug Development
May 7, 2019
EC Approves Akcea and Ionis’ Waylivra for Rare Lipid Disorder
Read More
Business
May 7, 2019
European Commission Approves BioMarin’s PKU Drug Palynziq
Read More
Rare Advocate
April 25, 2019
Rare Leader: Amanda Moore, CEO, Angelman Syndrome Foundation
Read More
Drug Development
April 25, 2019
Global Sales of Orphan Drug Expected to Grow at Twice the Rate of Non-Orphan Drugs
Read More
Business
April 24, 2019
Fulcrum Sees Promise in Failed Heart Drug as Treatment for FSHD
Read More
Legislation
April 24, 2019
ACE Kids Act to Improve Coordination of Care for Medically Complex Kids
Read More
Diagnosis
April 23, 2019
A Case for Whole Genome Sequencing
Read More
Drug Development
April 23, 2019
Report Predicts Sharp Jump in Clinical Development Productivity for Rare Disease Therapies
Read More
Drug Development
April 22, 2019
Novartis Investigating Patient Death in SMA Gene Therapy Trial
Read More
Research
April 22, 2019
Study Points to Lithium as Potential Treatment for Limb Girdle Muscular Dystrophy
Read More
Awareness
April 19, 2019
A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition
Read More
Rare Advocate
April 18, 2019
Rare Leader: Emily Milligan, Executive Director, Barth Syndrome Foundation
Read More
Drug Development
April 18, 2019
St. Jude Gene Therapy “Cures” Babies with “Bubble Boy” Disease
Read More
Uncategorized
April 17, 2019
When a Cold Is a Serious Threat
Read More
Business
April 17, 2019
FDA Grants Ultragenyx Rare Pediatric Disease Designation for Experimental Therapy
Read More
Business
April 16, 2019
Fibrocell Teams with Castle Creek on RDEB Gene Therapy
Read More
Business
April 16, 2019
NICE Gives Akcea Nod for Tegsedi to Treat Ultra-Rare hAATR
Read More
Business
April 9, 2019
FDA Notifies Zogenix of Inadequacies in Dravet Syndrome Drug Application
Read More
Business
April 9, 2019
Cyclerion Taps Shire’s Andreas Busch as Chief Innovation Officer
Read More
Business
April 8, 2019
Alnylam and Regeneron Enter Blockbuster Collaboration
Read More
Business
April 8, 2019
Audentes Expands Platform and Pipeline with Programs for DMD and DM
Read More
Business
April 5, 2019
How a Patient Group Helped Drive Drug Development in Rett Syndrome
Read More
Uncategorized
April 4, 2019
Rare Leader: Julie Raskin, Executive Director, Congenital Hyperinsulism International
Read More
Business
April 4, 2019
ICER Calls Spinraza Too Pricey, Urges Fair Pricing of Zolgensma
Read More
Business
April 4, 2019
FDA Grants Abeona Fast Track Designation for Sanfilippo Type B Gene Therapy
Read More
Uncategorized
Business
April 3, 2019
Dyne Therapeutics Raises $50 Million to Develop Therapies for Muscle Diseases
Read More
Business
April 3, 2019
EspeRare Partners with Dermelix to Develop Treatment for XLHED
Read More
Drug Development
April 2, 2019
Sangamo Provides Positive Updates on Hemophilia A and Beta Thalassemia Gene Therapy Trials
Read More
Business
April 2, 2019
AveXis Expands Gene Therapy Manufacturing Capacity with Acquisition
Read More
Business
April 1, 2019
EMA Grants Krystal Biotech PRIME Designation for DEB Gene Therapy
Read More
Business
April 1, 2019
FDA Grants AstraZenca and Merk’s NF1 Drug Breakthrough Therapy Designation
Read More
Drug Development
April 1, 2019
FDA Orphan Drug Designations Q1 2019
Read More
Rare Advocate
March 29, 2019
How Misperceptions Can Create Barriers to Care
Read More
Uncategorized
March 28, 2019
Rare Leader: Mary Anne Meskis, Executive Director, Dravet Syndrome Foundation
Read More
Business
March 28, 2019
Polyneuron Raises $22.6 Million to Advance Autoimmune Disease Therapies
Read More
Drug Development
March 28, 2019
Orchard Therapeutics Reports MLD Gene Therapy Shows Sustained Benefit
Read More
Awareness
March 28, 2019
Seeing Sickle Cell as a Blood Disease
Read More
Finance
March 27, 2019
Imara Raises $63 Million, Brings in Pfizer Rare Disease Head as CMO
Read More
Deals
March 27, 2019
ProQR and EB Research Form Drug Company
Read More
Research
March 27, 2019
NIH Gives $3.2 Million Grant to OMRF to Study Sarcoidosis
Read More
Drug Development
March 26, 2019
FDA Grants Promedior Breakthrough Therapy Designation for Idiopathic Pulmonary Fibrosis Drug
Read More
Drug Development
March 25, 2019
FDA Issues Draft Guidance on Natural History Studies for Rare Diseases
Read More
Business
March 22, 2019
Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs
Read More
Uncategorized
March 21, 2019
Rare Leader: Melissa Bryce Gamble, Executive Director, The Global Foundation for Peroxisomal Disorders
Read More
Business
March 21, 2019
FDA Approves Jazz Pharmaceuticals’ Narcolepsy Drug
Read More
Policy
March 21, 2019
Medicare Part D Spending on Specialty Drugs Climbs to $32.8B in 2015
Read More
Business
March 21, 2019
FDA Grants Enzyvant Fast Track and Rare Pediatric Disease Designations for Farber Disease Therapy
Read More
Business
Rare Advocate
March 20, 2019
Cure SMA and Parent Project Muscular Dystrophy Form Collaboration to Collect RWD
Read More
Business
March 20, 2019
Alexion Teams Up with Zealand, Affibody, in Pair of Development Deals
Read More
Business
March 20, 2019
Pfizer Takes Stake in Gene Therapy Developer Vivet
Read More
Business
March 20, 2019
GW Pharmaceuticals Sells Priority Review Voucher for $105 Million
Read More
Drug Development
March 18, 2019
Gottlieb Calls on Industry to Modernize Approach to Clinical Trials
Read More
Drug Development
March 15, 2019
Getting Misfolding Proteins to Shape Up
Read More
Rare Advocate
March 14, 2019
Rare Leader: Ashley Valentine, Co-Founder, Sick Cells
Read More
Rare Disease
March 13, 2019
A High School Student Unlocks A Rare Disease Mystery
Read More
Research
March 13, 2019
NDF Awards $700,000 in Grants for GNE Myopathy Research and Programs
Read More
Policy
March 12, 2019
Report Calls on NICE to Use Greater Flexibility in Reviewing Orphan Drugs
Read More
Business
March 8, 2019
A Different Approach to Inhibiting the Complement System
Read More
Rare Advocate
March 7, 2019
Rare Leader: Haley Oyler, President, SETBP1 Society
Read More
Policy
March 7, 2019
Patient Group Calls on BIO to Disavow “Price Gouging” by LEMS Drugmaker
Read More
Business
March 6, 2019
Alnylam Reports Positive Results from Late-Stage Study of Givosiran in AHP
Read More
Awareness
March 6, 2019
FDA to Hold Public Meeting on the Impact of Rare Diseases
Read More
Awareness
March 6, 2019
How Maria Voermans Found Her Calling
Read More
Drug Development
March 4, 2019
Oxford, Harrington Discovery Institute Form Center to Advance Rare Disease Therapies
Read More
Business
March 4, 2019
Biogen to Acquire Nightstar Therapeutics for $800 Million
Read More
Business
March 2, 2019
Perlara to Wind Down Operations
Read More
Awareness
March 1, 2019
Looking Back in Time to Find Rare Disease Patients Today
Read More
Awareness
February 27, 2019
A Rare Disease Gets Rock Star Treatment
Read More
Business
February 27, 2019
Amicus Establishes Global Research and Gene Therapy Center of Excellence
Read More
Deals
February 25, 2019
Roche Agrees to Acquire Spark Therapeutics for $4.8 Billion
Read More
Business
February 25, 2019
Ipsen to Acquire Clementia Pharmaceuticals for up to $1.3 Billion
Read More
Business
February 22, 2019
Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company
Read More
Policy
February 21, 2019
Catalyst Pharmaceuticals Defends Price of LEMS Drug in Letter to Senator Bernie Sanders
Read More
Awareness
February 21, 2019
Rare Leader: Connie Lee, President and CEO, Angioma Alliance
Read More
Business
February 21, 2019
Avexis Plans $60 Million Expansion of Manufacturing Facility in North Carolina
Read More
Awareness
February 21, 2019
Teaching Doctors When to See If a Horse Has Stripes
Read More
Diagnosis
February 20, 2019
Global Commission Releases Plan for Shortening Rare Disease Diagnostic Odyssey
Read More
Business
February 19, 2019
Nestle Health Sciences Exercises Option for Codexis PKU Treatment
Read More
Business
February 15, 2019
Passage Bio Raises $115.5 Million to Develop Gene Therapies for CNS Disease
Read More
Business
February 15, 2019
Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline
Read More
People & Organizations
February 15, 2019
Rare Leader: Lori Sames, CEO, Hannah’s Hope Fund
Read More
Drug Development
February 13, 2019
Study Questions the Value of Priority Review Vouchers as Incentives
Read More
Business
February 12, 2019
Neurogene Raises $68.5 to Advance Gene Therapies for Rare Neurological Diseases
Read More
Genomics
February 12, 2019
Combining Proteomics with Genomics Helps Pinpoint a Rare Disease
Read More
Innovation
February 12, 2019
Rare Disease Groups Launch Newborn Screening Study in Australia
Read More
Business
February 12, 2019
FDA Grants Clementia Rare Pediatric Disease Designation by FOP Drug
Read More
Awareness
February 8, 2019
Connecting Rare Disease Patients in India and the United States
Read More
Drug Development
February 8, 2019
Solid Biosciences Reports Disappointing Preliminary Results of DMD Gene Therapy Study
Read More
Drug Development
February 8, 2019
First Results of In Vivo Gene Editing Trial of MPS II Treatment Show Only Small Benefit
Read More
Business
February 7, 2019
FDA Approves Sanofi’s Cablivi for Rare Blood-Clotting Disorder aTTP, the First of a New Class of...
Read More
Rare Advocate
February 7, 2019
Rare Leader: Liz Marfia-Ash, President and Founder, GRIN2B Foundation
Read More
Awareness
Policy
February 5, 2019
Sen. Bernie Sanders Investigates $375,000 Price of LEMS Drug
Read More
Business
February 4, 2019
Alexion and Caelum Enter $550M Collaboration in AL Amyloidosis
Read More
Rare Disease
February 4, 2019
UK Grant Supports Diagnosis and Treatment of Rare Diseases
Read More
Innovation
February 4, 2019
ASCO Names Progress in Treating Rare Cancers Advance of the Year
Read More
Business
February 1, 2019
Regenerative Medicine Moves into the Spotlight
Read More

News

August 6, 2019
When the World Gets Dark, Kristin Smedley Shines a...
Read More
August 5, 2019
FDA Approves Daiichi Sankyo’s Turalio for Ra...
Read More
August 5, 2019
Rare Reading Before You Head to the 2019 RARE Pati...
Read More
July 31, 2019
Alizé Pharma 3 Raises $75 million to Advance Rare...
Read More
Read more

Contribute & Listen

Share Your Story
Be a Guest Blogger
Global Genes Rare cast
X