Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

With a progressive disease like FSHD, the loss of abilities and independence has an unpredictable timeline, says columnist.

Assistive Technology Development, or ATDev, is working with people with disabilities to develop systems for rehab and daily ...

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...

Background Over the last few years, there has been increasing attention to the involvement of the central nervous system in Duchenne muscular dystrophy (DMD). The aim of this study was to assess the ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Duchenne therapy may be evolving from slowing disease to reversing it—with RNA approaches offering the prospect of restoring function. Ongoing research will determine if these advances can deliver ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Dyne Therapeutics Inc. (NASDAQ:DYN) is one of the best small cap stocks with biggest upside potential. On August 4, Dyne Therapeutics announced that its investigational therapy, called DYNE-251, ...

- Data from the DELIVER registrational expansion cohort is expected in late 2025, with a potential BLA submission for U.S. accelerated approval anticipated in early 2026 - WALTHAM, Mass., Aug. 04, ...

A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular dystrophy, according to new research from the Smidt Heart Institute at ...