August 2025

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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Solid Biosciences announced a widely anticipated update on their INSPIRE DUCHENNE Phase 1/2 clinical trial of SGT-003, their investigational micro-dystrophin gene therapy for Duchenne muscular dystrophy. 15 participants have received SGT-003 so far across sites in the U.S., Canada, Italy, and the UK. No serious treatment-related adverse effects have been reported, and early safety results…

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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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A protein called MYOD, which is known to control the growth of muscle stem cells during muscle repair, has long been known to be able to turn on genes, but a recent study has now found that MYOD can also turn off certain genes. According to researchers, the findings may help pave the way toward…

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