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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 […]
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Oral therapy moves to follow-up after strong data in DMD adults
Dosing has begun in a long-term follow-up study testing Satellos Bioscience’s experimental oral therapy SAT-3247 […]
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PPMD Provides $400,000 in Funding to MyoGene Bio Through PPMD Venture Pathways Program to Support Development of Gene Editing Platform
PPMD and MyoGene Bio (MyoGene) are excited to announce that PPMD has provided $400,000 in […]
