Agamree, an approved steroid treatment for Duchenne MD, will be sold in Turkey by GEN, per an agreement with Santhera.

A protein called MYOD has long been known to be able to turn on genes, but a recent study has found that it also can turn off ...

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

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

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

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

All of those with FSHD are on unique paths, writes columnist Robin Stemple, but feedback from fellow patients is still immensely valuable.

View this post on Instagram Day 6 of #30daysofMD Introducing Brad Williams: A Scientist Tells His MD Story I first started to have muscle weakness at the age of 18. The first thing I noticed is that ...

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

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

View this post on Instagram Day 4 of 30 Days of MD Topic: Self Confidence and DMD This is Milan Patel’s story: Growing up with Duchenne Muscular Dystrophy, I was often self-conscious, especially after ...

View this post on Instagram Day 2 of 30 Days of MD Topic: Being a Teenager This is Kimberly Mullis’ story: Hey fam, I’m Kimmy!! (The one in the middle:) I have SMA II, and am truly living my best ...