Families say it's 'completely frustrating' that children with Duchenne Muscular Dystrophy are being denied access to free ...

Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.

The Jaffa Shriners’ DuBois Area Shrine Club continues to support a wheelchair program that provides essential equipment to ...

Key treatments for Alzheimer’s, rare cancers, and infectious diseases are under review, with new vaccines and therapies like ...

This year's Futures National Conference, hosted by CureDuchenne for the Duchenne and Becker MD communities, is scheduled to ...

Pfizer (NYSE:PFE) is a global pharmaceutical giant that has long been a cornerstone of medical innovation and today boasts a $128 billion market cap. Renowned for its blockbuster drugs like Lipitor ...

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic ...