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 ...

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 25 of #30DaysOfMD Introducing Gina Salminen- I wanted to share a few things about Cannabis and Facio Scapulo Humeral Muscular Dystrophy. I was diagnosed with ...

View this post on Instagram Day 10 of 30 Days of MD Topic: Medical Knowledge and MD This is @crystaleemarie’s story: I'm Crystal Rondeau and I have a form of MD called Spinal Muscular Atrophy Type 2.

View this post on Instagram Day 30 of 30 Days of MD Topic: Hoping for a Better Tomorrow This is Miroslava Zerova’s story: My name is Miroslava Zerova from Smolqn/ Bulgaria. l am 30 years old. I often ...

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 24 of #30daysofMD @brynnicorn “Well at least we know what it is”. Because in my 12 year old mind, I thought if we know what it was then we could fix it. I would not be ...