Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), ...

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 ...

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 ...

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

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 ...

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 ...

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

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 ...

The seasons are changing in central Nebraska. The leaves have turned yellow and orange, a beautiful contrast littered across the green lawns and familiar streets surrounding my home. The mornings are ...

The progression of myotonic dystrophy type 1, known as DM1, differs according to sex and age at symptom onset, according to the findings of a four-year study, but the researchers nonetheless reported ...