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Rare Disease Report®

The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.

Team Approach Needed for Advancing Treatments for Rare Diseases 

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

On the HCPLive Duchenne muscular dystrophy page, resources on the topics of medical news and expert insight into DMD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on the rare disease, and more.

Duchenne Muscular Dystrophy

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