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

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

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

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

Columnist Shalom Lim shares how his business, Rebirth Ensemble, will allow him to pursue visual arts and support artists with disabilities.

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 ...

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 ...

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 related neuromuscular conditions. For ...

A single low dose of the investigational gene therapy BB-301 helped with swallowing for the first two people with oculopharyngeal muscular dystrophy (OPMD) treated in a Phase 1b/2a clinical trial, ...

The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency — again maintained its recommendation not to renew conditional marketing authorization for Translarna ...