Articles

Podcasts

Videos

Rare Disease Report®

Minimal fat infiltration was observed on MR images from the SRP-9001 arm compared to participants from the natural history cohort.

New Gene Therapy Shows Promise Treating DMD

The approval is based on findings from a phase 2 clinical trial.

FDA Approves Viltolarsen for Duchenne Muscular Dystrophy

The RareWear program is designed to bridge medical device companies to rare disease patient applicants, seeking better disease and treatment monitoring.

Rare Genomics Institute Launches Free Rare Disease Device Program

New research suggests proposals aimed at reducing out of pocket costs may harm minimally-funded drug developers.

Medicare Part D Bills Could Harm Rare Disease Drug Development

The approval comes 4 months following a Complete Response Letter was submitted and successfully disputed, to Sarepta Therapeutics.

FDA Approves Golodirsen Injection for DMD with Genetic Mutations

The FDA has granted marketing authorization for the GSP Neonatal Creatine Kinase-MM to aid physicians in screening newborns for Duchenne Muscular Dystrophy.

FDA Authorizes Tool For Duchenne Muscular Dystrophy Screening in Newborns

There are only 4 case reports of this cancer in muscular dystrophy patients.  

Understanding a Rare Cancer in Duchenne Muscular Dystrophy Patients

Improvements in muscle and cardiac function were observed for 18 months in tested mice. 

Muscular Dystrophy Treatment with CRISPR Technology on the Way

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.

Cure CMD Co-Funds Research Grant With Muscular Dystrophy UK

Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.