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 ...

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 ...

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

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 ...

View this post on Instagram Day 28 of 30 Days of MD Topic: Genetics and Gardening This is @cocohollywood’s story: Lurking in my mom’s genetics is FSH muscular dystrophy (facioscapulohumeral muscular ...

View this post on Instagram Day 8 of #30daysofMD - Casey Duffield’s Story Accommodations and MD I think the word accommodation needs some accommodating. As someone who looks relatively healthy and ...