Enrollment is now ongoing in a first-in-human clinical trial testing EPI-321, a therapy designed to target the underlying ...

Columnist Shalom Lin and his partner, Amanda — both disabled artists — find that their challenges are exactly what can drive ...

The FDA has granted breakthrough therapy designation to DYNE-251 for the treatment of DMD in patients amenable to exon 51 ...

A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional ...

Sarepta will soon resume shipping Elevidys for ambulatory Duchenne muscular dystrophy patients after a review of safety data ...

Share this article: An experimental gene editing therapy for Duchenne muscular dystrophy (DMD) being developed by Precision Biosciences has received orphan drug status from the U.S. Food and Drug ...

Columnist Betty Vertin, whose days often feel overloaded, describes how it feels when her life as a caregiver is recognized and affirmed.

Elevidys (delandistrogene moxeparvovec-rokl), previously called SRP-9001, is a onetime gene therapy approved for certain patients with DMD.

A public workshop that columnist Shalom Lim and his girlfriend, Amanda, organized aimed to foster connection among people with disabilities.

With FSHD, even simple questions like "Would you like to go out to eat?" require many considerations, says columnist Robin Stemple.

Adeel Rizvi's philosophy is that life is a test assessing our ability to identify and cope with the journey, without forgetting about the surprise twists.