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

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

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

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

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 ...

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 ...