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They say the third time is the charm. However, as a mom to three sons with Duchenne muscular dystrophy (DMD), I don’t find that to be the case. I share seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie…

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What can you learn from the PPMD Adult Advisory Committee (PAAC) that you can’t ask Google? Honestly? A lot. Google can give you information, but it can’t tell you what it’s actually like to live with Duchenne or Becker muscular dystrophy. It can’t share the little workarounds, the “learned the hard way” moments, or the tips…

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Bridgebio is providing $100,000 to the Muscular Dystrophy Association (MDA) to advance projects aimed at improving multidisciplinary care for people with limb-girdle muscular dystrophy (LGMD). The awards will go to initiatives at Stanford Health Care and the University of Minnesota that aim to make LGMD care more coordinated, accessible, and flexible for patients across the…

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RegenXBio shared positive topline results from their pivotal Phase 3 AFFINITY DUCHENNE study of RGX-202 in 31 individuals with Duchenne aged 1 year and older.  The study met the primary endpoint with statistical significance, with 93% of participants achieving microdystrophin expression above 10%.  There was also a statistically significant correlation between microdystrophin expression and functional…

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REGENXBIO Inc. has announced positive topline and interim functional data from the pivotal Phase III portion of the Phase I/II/III AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne muscular dystrophy. RGX-202 is designed to deliver a microdystrophin via AAV8 through a one-time IV infusion.  The newly released data…

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Today, Gemma Biotherapeutics (GEMMABio), a clinical‑stage, global, genetic therapeutics company, shared information about the company’s Duchenne muscular dystrophy program, presenting preclinical data supporting candidate declaration for GB703, a novel, investigational gene therapy for the treatment of Duchenne.  GB703 utilizes a de-immunized, miniature transgene – a hybrid micro-utrophin/dystrophin that incorporates the self-utrophin-derived exon 8-11 region –…

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Joshua Vinson enjoys rain, camping, and cooking. Joshua works as a Quality Assurance Specialist in medical records processing. Their NMD diagnosis is Laing Distal Muscular Myopathy.  I think about fire a lot. I see the campfires I used to sit around before I grew too weak for weekend camping trips. I reminisce about the large…

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On May 1, my daughter Jill graduated from the University of Pittsburgh with a master’s degree in public policy and management. She also received the department’s student achievement award and was the student commencement speaker. Earning this degree took quite an effort, as she simultaneously worked full time as director of the Bureau of Policy,…

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Researchers at the University of Rochester are seeking adults with myotonic dystrophy type 2 (DM2) for a longitudinal natural history study (LEOPARD-DM2: Longitudinal Endpoint Optimization Providing an Assessment of Relevant Drugs in Myotonic Dystrophy Type 2). This study will collect health information through period assessments over 2.5 years. Findings from this study could help researchers better…

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PPMD recently hosted a webinar centered on common questions from families: is exercise recommended in dystrophinopathy care and how it can be done safely? Experts discussed what we currently understand, what researchers are still exploring, and how this information can be thoughtfully incorporated into day-to-day life. Speakers: Claudia Senesac, PT, DPT Donovan Lott, PT, PhD,…

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