Corticosteroid-related growth stunting in Duchenne muscular dystrophy might be partly reversed by switching to the medication Agamree, data suggest.

Columnist Betty Vertin has begun doing three things to give herself time to recover from the business of her life as a Duchenne caregiver.

Columnist Robin Stemple's latest excursion was a trek across the city to discover where to buy a lift chair for his ...

Sarepta plans to ask the FDA for full approval of exon-skipping Duchenne muscular dystrophy treatments Amondys 45 and Vyondys 53.

Bridgebio Pharma asked the FDA to approve BBP-418, an oral therapy that could be the first treatment for LGMD2i.

Early trial data suggest RNA therapies SRP-1001 and SRP-1003 reach muscle and show biomarker activity in muscular dystrophy.

Shalom Lim is a University of Liverpool graduate, having completed an honors bachelor’s degree in criminology and security in July 2021. Born with Duchenne muscular dystrophy and diagnosed at 4 months ...

Histone deacetylase inhibitors are a treatment approach designed to work by blocking an enzyme that turns off gene activity. This may ultimately help by slowing muscle loss and disease progression.

Exon skipping is a treatment approach for people whose Duchenne muscular dystrophy is due to certain mutations in its causative gene. Exon skipping works like a molecular patch, so that the DMD gene ...

“Take him home and give him a good life.” This was the main advice given to Jessica and Chris Curran by a genetic counselor after their son, Conner, was diagnosed with Duchenne muscular dystrophy (DMD ...

Muscular dystrophy refers to a group of genetic diseases that cause progressive muscle weakness and loss. There are more than 30 types of muscular dystrophy, all with different causes and symptoms.

Rob Stemple is a lifelong advocate for people with disabilities. He was diagnosed with FSHD in 1971 at age 14. Rob struggled with its’ affects for over 50 years. He lost his eyesight in a devastating ...