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

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

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

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

I’m currently continuing to do the exercises my physical therapist recommended during my last therapy session. However, it hasn’t been stopping the progression of my facioscapulohumeral muscular ...

People with FSHD, ages 16 to 70, enrolled in this Phase 1/2 FORTITUDE trial cohort will aid biomarker data to support early approval request.