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Dear PPMD Community, For more than thirty years, I have had the honor of standing beside you—parents, families, clinicians, researchers, and advocates—united in a single purpose: to end Duchenne and Becker muscular dystrophy, and to ensure that every child and adult in our community has access to the very best care and therapies. Today, I’m…

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Para-dressage athlete, Riley Garrett Riley Garrett has found her passion and purpose as a para-dressage competitor and through coaching and empowering other young athletes to challenge themselves and pursue their dreams. Born with Bethlem myopathy, the 25-year-old elite equestrian athlete is already a national and international standout in the para-equestrian dressage arena. And now she…

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The steroid therapy Agamree (vamorolone), which is used for helping to preserve muscle function in people with Duchenne muscular dystrophy (DMD), will be sold and promoted in Turkey by Gen İlaç ve Sağlık Ürünleri San. ve Tic. A.Ş. (GEN), per an agreement with Santhera Pharmaceuticals. “As leaders in the Turkish specialty pharmaceuticals market, GEN were…

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Every year, thousands of newborns in the U.S. are screened for serious conditions whose early detection can change the course of their lives. Right now, we have a chance to ensure Duchenne muscular dystrophy is one of them. The U.S. Department of Health and Human Services (HHS) is currently reviewing the nomination of Duchenne to…

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In this webinar, Dr. Naz Dastgir, Executive Medical Director and Clinical Development Lead for RGX-202 at REGENXBIO, shared information on the active and enrolling AFFINITY DUCHENNE trial and how families can obtain additional information about RGX-202 and potential clinical trial participation. Watch HERE The post REGENXBIO AFFINITY DUCHENNE: Trial of Investigational RGX-202 Gene Therapy appeared first…

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Back to School with Duchenne: Navigating this new 2025 school year is a challenge for anyone. But with a condition like Duchenne muscular dystrophy to contend with, back to school can become even more difficult. To find success while transitioning back to school, it’s important to communicate with teachers, administrators, and staff your needs. That’s…

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Solid Biosciences has shared an update on the company’s Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003 for the treatment of individuals living with Duchenne. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their novel AAV capsid designed to increase skeletal and cardiac muscle targeting while detargeting the…

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I’m a musician, and so are my brothers, Ron and Tim. We’ve all played professionally, but it’s been a long time since we’ve played together. That changed last month, but let me explain why it was such an event. Back in the late ’70s we were part of the Frank Kalik Orchestra, a Polka Hall…

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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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Madison is from Baton Rouge, Louisiana, and lives with congenital muscular dystrophy. She is an upcoming senior at St. Joseph Academy. She has been a dancer since she was 3 years old and has a passion for using her voice to advocate for the neuromuscular disease community. High school can be challenging but being a…

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