Duchenne Muscular Dystrophy

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.

DMD Treatment Granted Orphan Drug & Rare Pediatric Disease Designations

August 16, 2018

Article

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).

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