Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...

Columnist Robin Stemple says he chooses to focus on what FSHD progression hasn't taken from him. The small victories matter, ...

Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...

Modalis Therapeutics is seeking to make MDL-101 the first treatment for people with LAMA2-related congenital muscular ...

Columnist Betty Vertin wishes she could take away the pain over the milestones in the teenage years her sons with DMD will miss.

Betty Vertin is a mother and writer living in rural Hastings, Nebraska, with her husband and seven children. Betty is a caregiver to three sons, Max, Rowen, and Charlie. Her oldest was diagnosed with ...

Three weeks ago, my girlfriend, Amanda, and I embarked on a unique journey by co-founding Rebirth Ensemble (RE), an art-making business with a distinct purpose. This partnership is deeply rooted in ...

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 ...

A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran (del-brax), an experimental and potentially disease-modifying therapy for facioscapulohumeral muscular dystrophy ...