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Duchenne Muscular Dystrophy
FDA Approves Viltolarsen for Duchenne Muscular Dystrophy
August 12, 2020
Article
The approval is based on findings from a phase 2 clinical trial.
Rare Genomics Institute Launches Free Rare Disease Device Program
February 29, 2020
Article
The RareWear program is designed to bridge medical device companies to rare disease patient applicants, seeking better disease and treatment monitoring.
Medicare Part D Bills Could Harm Rare Disease Drug Development
February 29, 2020
Article
New research suggests proposals aimed at reducing out of pocket costs may harm minimally-funded drug developers.
FDA Approves Golodirsen Injection for DMD with Genetic Mutations
December 13, 2019
Article
The approval comes 4 months following a Complete Response Letter was submitted and successfully disputed, to Sarepta Therapeutics.
FDA Authorizes Tool For Duchenne Muscular Dystrophy Screening in Newborns
December 12, 2019
Article
The FDA has granted marketing authorization for the GSP Neonatal Creatine Kinase-MM to aid physicians in screening newborns for Duchenne Muscular Dystrophy.
Understanding a Rare Cancer in Duchenne Muscular Dystrophy Patients
January 25, 2019
Article
There are only 4 case reports of this cancer in muscular dystrophy patients.
Muscular Dystrophy Treatment with CRISPR Technology on the Way
January 17, 2019
Article
Improvements in muscle and cardiac function were observed for 18 months in tested mice.
Cure CMD Co-Funds Research Grant With Muscular Dystrophy UK
September 18, 2018
Article
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.
Top 5 Rare Disease News of the Week—September 2, 2018
September 08, 2018
Article
Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.
CRISPR Gene Editing Treatment for Duchenne Muscular Dystrophy Moves Closer to Clinical Trials
September 04, 2018
Article
The latest approach to developing a gene therapy for Duchenne muscular dystrophy shows promising results.