Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals ...

The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.

The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to NS-051/NCNP-04, which is ...

This follows confirmation that no appeals were received against the Final Draft Guidance (FDG) recommendation announced on December 10, 2024. Following this, Santhera has already started launch ...

Undervalued biotech company Regenxbio Inc. shows promise with cutting-edge gene therapy technology for AMD and MPS II. Click ...

Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular ... dystrophy (DMD) community and prompted layoffs at Pfizer. Investigators said the unique features ...

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscle ... 2008). Despite their distinct clinical features, FSHD and BMD exhibit a wide range of overlapping phenotypic spectrum, ...

Credit: Dan76 / Shutterstock. Percheron Therapeutics has announced that it will investigate avicursen (ATL1102) in other indications besides Duchenne muscular dystrophy (DMD) following disappointing ...

Delpacibart zotadirsen is under clinical development by Avidity Biosciences and currently in Phase II for Duchenne Muscular Dystrophy. According to GlobalData, Phase II drugs for Duchenne Muscular ...

1 The increased production of a functional protein may be associated with a clinical benefit ... FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation. News release. US Food ...

to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. The company noted that the submission of the BLA marks a pivotal step for Capricor and those impacted by DMD. “This ...