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Welcoming the New Members of PAAC for Fall 2025
PPMD’s Adult Advisory Committee (PAAC) recently expanded with 19 inspiring new members, bringing the committee
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Exoskeletons Improve Movement for Those with Neuromuscular Diseases
Exoskeletons are wearable devices that can provide physical support and improve functional mobility for people
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DMD drugs show promise despite trial’s missing main goal: Sarepta
Exon-skipping therapies Amondys 45 (casimersen) and Vyondys 53 (golodirsen) show “positive and encouraging trends” for people
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Updates on Entrada’s exon skipping clinical trials
As an early investor in Entrada Therapeutics, we are pleased to share their progress on
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Advancing Brain and Behavior Care in Dystrophinopathy
Recently, PPMD led a meeting with experts from across the US and Europe to discuss
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Guest Voice: Caregiving isn’t easy, but it’s not a burden
We had only been married a few months when my husband woke me in the
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Finding balance as I manage my DMD-associated cardiomyopathy
Turning 30 last month was a significant milestone in my Duchenne muscular dystrophy (DMD) journey.
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Solid Biosciences Shares Interim Data from INSPIRE DUCHENNE Trial of SGT-003
Solid Biosciences Inc. has announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE
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Understanding Fat Embolism Syndrome (FES) and Protecting Bone Health
PPMD recently hosted a webinar about Fat Embolism Syndrome (FES), covering what FES is, what
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How Expanded Access and Compassionate Use Broaden Access to Investigational Therapies
In 2012, Arturito Estopinan was diagnosed with thymidine kinase 2 deficiency (TK2d) at 17 months
