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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
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REGENXBIO Shares Positive Interim Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202
REGENXBIO has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of
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Solid Biosciences Shares Interim Positive Update on Phase 1/2 INSPIRE DUCHENNE Trial
Solid Biosciences Inc. has shared updated positive interim data from the ongoing Phase 1/2 INSPIRE
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ITF Therapeutics Shares Community Letter and Presents New Data and Analyses on DUVYZAT® (givinostat)
ITF shared a community letter and presented new data, including long-term safety observations based on data
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Solid Biosciences Provides Positive Interim Clinical Update from Phase 1/2 INSPIRE DUCHENNE Trial
SGT-003, an investigational microdystrophin gene therapy for Duchenne, continues to demonstrate an encouraging safety profile
