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Rare Daily Staff The nonprofit EveryLife Foundation for Rare Diseases has appointed Michael Pearlmutter into the new […]
Read moreStoke Raises $125 Million After Reporting Positive Results from Dravet Study
Rare Daily Staff Stoke Therapeutics tapped the public market to raise $125 million after reporting positive results […]
Read moreBuying and Building a Gene Therapy Presence
Ha Tran, medical head of cell and gene therapy for Astellas Pharma, discusses the company’s vision for […]
Read moreFDA Approves Merck’s Winrevair for Adults with Pulmonary Arterial Hypertension
Rare Daily Staff The U.S. Food and Drug Administration Merck’s Winrevair for the treatment of adults with […]
Read moreStoke Surges After Reporting Positive Results from Dravet Syndrome Study
Rare Daily Staff Stoke Therapeutics’ shares surged 90 percent after the company reported positive new data from […]
Read moreFDA Grants Fast Track Designation to J&J’s Nipocalimab to Reduce Risk of FNAIT in Alloimmunized Pregnant Adults
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation for Johnson & Johnson’s […]
Read moreTUDCA-ALS Late-Stage Trial in ALS Patients Fails
Rare Daily Staff The TUDCA-ALS consortium said top-line results from the European phase 3 clinical trial of […]
Read moreResearchers Unlock Genetics of Ultra-Rare Neurodevelopmental Disorder
Rare Daily Staff Researchers at Ottawa’s Faculty of Medicine have revealed how gene mutations work to cause […]
Read moreCelebrating the Most Expensive Drug for the Disease You Never Heard Of
When doctors at Children’s Hospital of Philadelphia diagnosed Maria Kefalas’ 2-year-old daughter Cal in 2012 with metachromatic […]
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