Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Longboat Key firefighters “filled the boot” through a fundraising effort for the Muscular Dystrophy Association.

Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...

"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne Muscular Dystrophy Cara Lynn Shultz is a writer-reporter at PEOPLE. Her ...

Parent Project Muscular Dystrophy (PPMD), in partnership with the Foundation to Eradicate Duchenne (FED), today announced Representative Troy Balderson (OH-12), Senator Susan Collins (ME), Senator Amy ...

HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 -- 48-week trial will enroll approximately 150 ...

Shares of Dyne Therapeutics gained after the company shared positive data from a continuing trial of a potential Duchenne muscular dystrophy treatment. The stock rose 14%, to $16.93, midday Monday.

Muscular dystrophy is a term which comprises a diverse group of genetic disorders that cause weakness and degeneration of skeletal muscle due to defective proteins critical for muscle structure and ...

The timing of the partial hold is “odd,” according to analysts at Stifel, who noted that the preclinical data the FDA took ...

A test of strength that raises money for a good cause. Sunday was New Bern Fire and Rescue’s annual firetruck pull.Teams of ...

SACRAMENTO (CBS13) — Comedian, actor and filmmaker, Jerry Lewis left behind decades worth of precious moments and laughs through his work on the big screen and in television, but it was his devotion ...