Decker was given the 2025 MDA Legacy Award, recognizing his efforts to advance gene therapy, scientific research, and the ...

Use of DYNE-101 is leading to improvements in muscle, and specifically finger, function in myotonic dystrophy type 1 patients ...

Columnist Patrick Moeschen believes it's important to talk about what makes life fulfilling. For him, it's been a life of ...

Columnist Betty Vertin says her youngest daughter is starting to become aware of her older brothers' health challenges with DMD.

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, per interim data.

Columnist Shalom Lim is sad to say goodbye to his caregiver of 18 years, but looks forward to the new chapter of life this ...

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...

Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males with Duchenne muscular dystrophy (DMD), according to data from the ...

Capricor Therapeutics’ investigational therapy deramiocel, formerly CAP-1002, which is being considered for approval in the U.S., continued to show an ability to preserve upper limb function in boys ...