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The U.S. Food and Drug Administration has granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping Duchenne muscular dystrophy (DMD) treatment from NS Pharma. Treatments granted this designation are aimed at serious or life-threatening diseases affecting children and fewer than 200,000 U.S. patients. “We are grateful for this designation, which can help us accelerate the…

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Atamyo Therapeutics has partnered with the Dion Foundation for Children with Rare Diseases to expand into the U.S. a clinical trial of ATA-200, a potential gene therapy for limb-girdle muscular dystrophy (LGMD) type 2C/R5 — dubbed LGMD2C/R5. The Phase 1b study (NCT05973630), designed to evaluate the safety, efficacy, and pharmacological and immunological properties of ATA-200,…

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Recently, PPMD’s Alexis Hazlett was joined by panelists Jodi Krause (Colorado Children’s Hospital), Jill Castle, and Patrick Moeschen to discuss advocating for your child throughout the school year. The speakers dove into the differences between IEP (Individualized Educational Plan) and 504 plans, understanding your child’s educational rights, and shared tips to enhance communication and working…

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As most of America’s children go back to school, I thought it’d be timely to offer readers my perspective, as a retired teacher and wheelchair user, on the beginning of an academic year. I began teaching middle school music and band in the fall of 1995. I was 22 years old and had just graduated…

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While observing World Duchenne Awareness Day over the weekend, I reflected on the journey my family and I have taken, which has been shaped by Duchenne muscular dystrophy (DMD). This year the Muscular Dystrophy Association (Singapore), or MDAS, celebrated the day during its annual Go the Dystance carnival. Growing up with DMD, I found that…

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I walked home from school today. I’m almost 45 years old, yet today, with my backpack on, my empty lunch container stuffed inside, and a sweater tied around my waist, I walked home from school. As I pondered whether I was overthinking that image, my daughter Mary, 9, who goes to the same school where…

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Up to a year of treatment with DYNE-251, Dyne Therapeutics’ investigational exon 51-skipping therapy, led to improvements in motor function for boys with Duchenne muscular dystrophy (DMD), according to a clinical trial update. Based on these positive data from the Phase 1/2 DELIVER clinical trial (NCT05524883), Dyne is launching registrational groups of trial participants, the…

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On Thursday, September 5, 2024, PPMD hosted a special webinar, “PPMD Advocacy: Raising Our Voices in 2024 and Beyond,” bringing together advocates to reflect on PPMD’s advocacy efforts in our 30th anniversary year and discuss how we continue to drive policy change for those living with Duchenne and Becker muscular dystrophy. Moderated by PPMD’s Senior…

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SEE THE FULL PRESS RELEASE BELOW AND HERE – Second annual DREAMS Grant Program awards $25,000 each to three U.S.-based non-profit organizations working to achieve greater equality for those living with Duchenne – – Announced in celebration of World Duchenne Awareness Day, grants are designed to fund efforts advancing diversity, equity, inclusion and accessibility within…

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PPMD is dedicated to fighting for every future as we strive to end Duchenne. Since 2011, PPMD has been a proud supporter of the Myology Course, presented by Nationwide Children’s Hospital (NCH) and The Ohio State University which aims to educate and advance the next generation of neuromuscular physicians and scientists. This commitment is essential…

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