Agamree (vamorolone) was recommended for use in the NHS in England, Wales, and Northern Ireland for treating Duchenne ...

I’m more than a caregiver, but it took a recent life change for me to realize that. I’m a mom to seven children I share with my husband, Jason, including three of our sons — Max, 19, Rowen, 15, and ...

FSHD exacts a toll on columnist Robin Stemple when he is out and about. He calls these symptom flares "FSHD hangovers." ...

Eight $250,000 prizes will be awarded for key milestones in FSHD treatments and $8 million will be given for showing clinical ...

Arrakis' oral therapies targeting disease-driving RNA in myotonic dystrophy have shown promise in preclinical studies.

Columnist Shalom Lim describes a moving performance at the Enabling Lives Festival in Singapore, an example of the power of ...

Sarepta has acquired the global rights to develop ARO-DUX4 and ARO-DM1, two RNAi therapeutic candidates for types of muscular ...

A woman who developed heart failure due to Duchenne muscular dystrophy (DMD) was diagnosed and treated before she got pregnant, which led to a healthy outcome for her and her baby girl, according to ...