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Sarepta trial enrolling for ELEVIDYS treatment with enhanced immunosuppression in non-ambulant individuals
Sarepta therapeutics is seeking to enroll 25 non-ambulant individuals with Duchenne in a clinical trial
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MDA Ambassador Guest Blog: Navigating a Busy Life with a Feeding Tube
Richard Farrell Jr. is 19 years old and lives in Pennsylvania. He was diagnosed with
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MDA 2026: SGT-003 gene therapy shows early promise for DMD in trial
SGT-003, an investigational gene therapy for Duchenne muscular dystrophy (DMD), worked as expected to increase levels
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Finding time for some quiet thoughts amid the travel and doctor visits
I’m one of those people who can get lost deep in thought. As a busy
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Community Comes Together on Capitol Hill: A Recap of PPMD’s 2026 Advocacy Conference
Each year, the Duchenne and Becker muscular dystrophy community comes together in Washington, DC with
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MDA 2026: Early trial of PGN-EDODM1 shows biomarker activity in DM1
PGN-EDODM1, Pepgen’s experimental therapy for myotonic dystrophy type 1 (DM1), was generally well tolerated in
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Capricor Therapeutics Announces Positive Data from HOPE-3 Study of Deramiocel
Capricor Therapeutics has announced additional analyses and new functional outcomes data from the Phase 3
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MDA 2026: Sevasemten halts functional decline in Becker trial
Becker muscular dystrophy (BMD) patients who received the experimental treatment sevasemten in clinical trials had
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As my FSHD progression worsens, I’m battling feelings of shame
My facioscapulohumeral muscular dystrophy (FSHD) has progressed to the point where daily activities are increasingly
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REGENXBIO Reports New Positive Interim Data from Phase I/II AFFINITY DUCHENNE Gene Therapy Trial
REGENXBIO GENE THERAPY RGX-202, an investigational gene therapy for Duchenne, continues to show a favorable
