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

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

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 ...