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Calling All Adults with Duchenne & Becker: Join the 2025 PPMD Adult Advisory Committee
Have you been looking for a way to engage with the community more? Do you have a personal experience that you would like to share with the larger community or through federal and state advocacy efforts? Are you interested in serving as a mentor to others with Duchenne and/or Becker? PPMD is seeking adult (ages…
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The missed milestones in the teenage years because of DMD
I have three teenage sons — Max, 18, Rowen, 15, and Charlie, 13 — who have Duchenne muscular dystrophy (DMD). They have met several sweet milestones over the years, and I love celebrating them. I’ve always made a big deal about birthdays, for instance, as well as decorating the house on the night of the first…
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Solid Biosciences Shares Updates on INSPIRE DUCHENNE Clinical Trial of SGT-003
Yesterday, Solid Biosciences shared new updates on the INSPIRE DUCHENNE clinical trial for their next-generation AAV micro-dystrophin gene therapy candidate, SGT-003, for the treatment of Duchenne. Based on early study results, the trial has expanded its enrollment to a total of 43 participants and broadened the age range to include individuals from 4 to less…
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Patients with certain mutations lose their walking ability earlier: Study
Specific genetic mutations in Duchenne muscular dystrophy (DMD) influence how long patients retain the ability to walk, even when treated with corticosteroids, according to a study that highlights the importance of genetic testing in predicting disease progression. These findings are relevant because understanding how fast the disease progresses based on different genetic profiles could help doctors…
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REGENXBIO Shares Updates on Phase I/II AFFINITY DUCHENNE® trial of RGX-202
Yesterday, REGENXBIO shared important updates on their Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for Duchenne during their third quarter financial results update. RGX-202 utilizes a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide benefit in place of the missing full-length dystrophin protein. Key highlights of…
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Supporting artists with disabilities through a new business venture
Three weeks ago, my girlfriend, Amanda, and I embarked on a unique journey by co-founding Rebirth Ensemble (RE), an art-making business with a distinct purpose. This partnership is deeply rooted in our shared passion for visual art, which has been a source of joy but also a progressively tricky challenge due to her visual impairment…
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I always try to pause to appreciate the small victories
I’m currently continuing to do the exercises my physical therapist recommended during my last therapy session. However, it hasn’t been stopping the progression of my facioscapulohumeral muscular dystrophy (FSHD). While I’m hoping the exercises are slowing the progression down, it seems like it gets harder every day for me to move. This relentless progression has…
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Sarepta Announces Discontinuation of SRP-5051 Development for Duchenne
Sarepta Therapeutics today announced that the company is discontinuing its SRP-5051 (vesleteplirsen) development program, including the global, Phase 2, multi-arm, ascending dose MOMENTUM study. Vesleteplirsen is an investigational, next-generation treatment utilizing Sarepta’s PPMO chemistry and exon-skipping technology for individuals with Duchenne amenable to exon 51 skipping. According to Sarepta, this decision was informed by information…
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Phase 1/2 trial of del-brax enrolling new, possibly pivotal FSHD group
A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran (del-brax), an experimental and potentially disease-modifying therapy for facioscapulohumeral muscular dystrophy (FSHD), its developer, Avidity Biosciences, announced. The additional trial cohort, which is expected to be recruited in full early next year, aims to evaluate the effects of del-brax on…
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WATCH: Exploring Muscle Regeneration – A New Approach with Satellos (Webinar Recording)
Satellos recently joined PPMD for a community webinar exploring a new approach for the treatment of Duchenne. Satellos presented on the discovery behind SAT-3247, a small molecule drug that mobilizes the body’s own muscle stem cells to repair and regenerate muscles. The session also includes a Q&A portion where Satellos addresses questions from the community…
