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

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

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

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.

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 ...