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February 29, 2020
The RareWear program is designed to bridge medical device companies to rare disease patient applicants, seeking better disease and treatment monitoring.
New research suggests proposals aimed at reducing out of pocket costs may harm minimally-funded drug developers.
December 13, 2019
The approval comes 4 months following a Complete Response Letter was submitted and successfully disputed, to Sarepta Therapeutics.
December 12, 2019
The FDA has granted marketing authorization for the GSP Neonatal Creatine Kinase-MM to aid physicians in screening newborns for Duchenne Muscular Dystrophy.
January 25, 2019
There are only 4 case reports of this cancer in muscular dystrophy patients.
January 17, 2019
Improvements in muscle and cardiac function were observed for 18 months in tested mice.
September 18, 2018
Cure CMD (Congenital Muscular Dystrophy) is pleased to announce the recipient of a grant co-funded with Muscular Dystrophy UK: Dr. Giséle Bonne at the INSERM-Myology Institute in Paris.
September 08, 2018
Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.
September 04, 2018
The latest approach to developing a gene therapy for Duchenne muscular dystrophy shows promising results.
August 16, 2018
The FDA has granted an orphan drug designation and a rare pediatric disease designation to Wave Life Sciences Ltd.’s WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD).