The big pharma company will no longer progress emugrobart to late-stage trials in FSHD and SMA due to a lack of efficacy.

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

The main beneficiary of Roche’s discontinuation of an investigational spinal muscular atrophy drug is Scholar Rock, which was hobbled by manufacturing concerns at a Novo Nordisk facility last year but ...

While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

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.

The Ironman In You project aims to raise awareness on muscular dystrophy It also aims to raise money for members of the Muscular Dystrophy Association of Singapore As of June 25, this project had ...

Team Soph’s Mighty Striders tackle Glasgow Kiltwalk to raise £8000 for Muscular Dystrophy UK, honouring Sophie Hutchison and raising LGMD awareness.

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window Eight therapies ...