The FDA has given the green light to a clinical trial testing ATA-200, a gene therapy for limb-girdle muscular dystrophy ...
Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), ...
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.
Columnist Betty Vertin, the mother of three boys with DMD, has the experience and self-assurance to know she can do hard things.
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