NEXT: James Valentine of Hyman,Phelps & McNamara Discusses the Future of Rare Disease
October 20, 2019
Related
EveryLife Foundation for Rare Diseases Welcomes Michael Pearlmutter as CEO
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 […]
Read more