Duchenne Muscular Dystrophy

FDA Approves Viltolarsen for Duchenne Muscular Dystrophy

August 12, 2020


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

Rare Genomics Institute Launches Free Rare Disease Device Program

February 29, 2020


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


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


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


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


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


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


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


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


The latest approach to developing a gene therapy for Duchenne muscular dystrophy shows promising results.