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Watch: Avidity Biosciences – Topline Data from EXPLORE44® Clinical Trial (Webinar Recording)
Avidity Biosciences recently joined PPMD for a community webinar to share topline data from their
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MDA 2025: Benefits seen for 3 OPMD patients given gene therapy
The use of experimental gene therapy BB-301 led to improvements in swallowing ability for the
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Simply Stated: Updates in Neuromuscular Junction (NMJ) Disorders
The neuromuscular junction (NMJ) disorders are a group of conditions that disrupt the communication between motor
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Clinical Research Opportunity: LION-CS101 a Phase 1/2 Study of AB-1003 in Adults with LGMD2I/R9
Researchers at AskBio are seeking adults with genetically confirmed limb-girdle muscular dystrophy (LGMD) type 2I/R9 to
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Dr. Peter Marks Resigns from FDA Center for Biologics Evaluation and Research
PPMD is deeply disappointed that Dr. Peter Marks has made the decision to leave his
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Pro Tips to Add More Movement to Your Day
Adding more movement or physical activity to each day is a common goal among people
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MDA 2025: Duvyzat delays walking loss, lessens lung function decline
Long-term treatment with Duvyzat (givinostat) may help delay the loss of walking ability and lessen
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Wave Life Sciences Announces Positive Data from FORWARD-53 Clinical Trial
Wave Life Sciences has announced encouraging interim results from its Phase 2 FORWARD-53 clinical trial
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A virtual art show gave FSHDers a chance to show off our talents
I was tempted to do a play on the show “America’s Got Talent” and title
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Target to repair injured muscles discovered
A team has discovered a possible therapeutic approach to repair injured muscles either from aging
