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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 with Duchenne muscular dystrophy (DMD), even though top-line results from a Phase 3 trial testing the treatments showed the study failed to meet its main goal, developer Sarepta Therapeutics said. Results from the Phase 3 ESSENCE clinical trial may have been…
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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 their exon-skipping programs in Duchenne. For Exon 44: Entrada completed enrollment for the first cohort of the ex-US ELEVATE-44-201 study for those amenable to skipping exon 44, and are on track to report data in Q2 2026. They expect to…
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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 the evolving understanding of brain and behavior implications in individuals with dystrophinopathy. The two day workshop, led by Drs. Molly Colvin (Massachusetts General), Natalie Truba (Nationwide Children’s Hospital) and Francesco Muntoni (University College London), focused on how dystrophin impacts brain…
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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 middle of the night to help him reposition. Half-asleep and irritated, I got up and muttered in the darkness, “I don’t want a husband anymore.” I didn’t remember saying it until he told me the next morning. Back then, we…
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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. For years, my care has revolved around keeping my vital functions strong enough to sustain a body whose muscles continue to wither with every passing second. Having lived with DMD since birth, I’ve witnessed firsthand how the disease stresses and…
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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 clinical trial and provided an update on its planned meeting with the U.S. Food and Drug Administration (FDA) to discuss potential registrational pathways for SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin…
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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 symptoms to watch for, and how to help keep people with dystrophinopathy safe and healthy. Four clinical experts shared practical tips on preventing falls, how to support bone health and decrease the risk of fractures, recognizing signs of FES after…
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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 old. This extremely rare form of mitochondrial myopathy causes weakness in the limbs, as well as trouble breathing and swallowing. At the time, there was no approved treatment for TK2d, but his parents, Olga and Art, learned of an investigational…
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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 in men with Duchenne muscular dystrophy (DMD). The study — called LT-001 (NCT06867107) — enrolled five patients who completed a previous Phase 1 trial (NCT06565208), where SAT-3247 was shown to improve measures of muscle function after about a month of treatment.…
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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 funding to MyoGene through PPMD Venture Pathways, the organization’s venture-philanthropy initiative that provides industry funding to accelerate therapeutic development for Duchenne and Becker. This funding commitment aims to advance the development of MyoGene’s gene editing strategy and potentially leverage additional…
