Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...
Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...
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.
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