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DMD gene therapy leads to sustained benefits for 3 boys in trial
Three boys with Duchenne muscular dystrophy (DMD) who were given the experimental gene therapy GNT0004 in a clinical trial were seeing sustained improvements in motor function and reductions in markers of muscle damage two years after the one-time treatment. Genethon, GNT0004’s developer, presented the findings at the European Society of Gene and Cell Therapy (ESGCT)…
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Avidity Biosciences Announces Positive Pre-BLA Meeting with FDA for del-zota
Avidity Biosciences, Inc. has announced the completion of a positive pre-Biologics License Application (BLA) meeting with the U.S. Food and Drug Administration (FDA) regarding its upcoming BLA submission of delpacibart zotadirsen (del-zota) for the treatment of Duchenne in those amenable to exon 44 skipping. Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) conjugated with…
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My sons with Duchenne are each other’s best friends, and that’s OK
Moms worry about their children. As a mother of seven — Lexi, 24; Max, 19; Chance, 18; Rowen, 16; Charlie, 14; Mary, 11; and Callie, 3 — I know firsthand what it’s like to be anxious and concerned about kids. My children’s ages span 20 years — another thing to worry about. Some are navigating…
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Standing Together for Access: Ensuring Duchenne Patients Can Benefit from Approved Therapies
PPMD has always fought to ensure that every individual living with Duchenne has access to safe, effective, FDA-approved treatments. This week, PPMD joined six partner organizations: Best Day Ever Foundation, Charlie’s Cure, CureDuchenne, Little Hercules Foundation, Muscular Dystrophy Family Foundation, and Team Joseph, to urge New York State to reconsider the recent Drug Utilization Review…
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Agamree becomes 1st approved DMD treatment in Canada
Health Canada has approved Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older, making Agamree the first approved DMD treatment in Canada. Canada’s approval is the sixth for Agamree to treat DMD. The therapy is approved in the U.S., the U.K., and the European Union, as well as in China…
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MDA Ambassador Guest Blog: How Adaptive Sports Changed My Life
Brayden is 17 years old and has been playing wheelchair basketball since he was 8. He is also a wheelchair track and field athlete. Brayden lives with Charcot-Marie-Tooth (CMT) Type 1A. He first became an MDA Ambassador when he was 5 years old and he loves supporting MDA’s mission. Brayden is consistently on the honor…
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Catch Up on World Muscle Society 2025
Each year, the World Muscle Society (WMS) Annual Congress brings together the best minds in neuromuscular research, clinical care, patient advocacy, and industry. The meeting offers a dynamic platform for sharing new data, fostering collaborations, and advancing the science behind therapies for diseases like Duchenne and Becker muscular dystrophy. This year’s event in Vienna, Austria,…
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Disability advocates let their voices be heard at symposium
A couple weeks ago, I co-emceed the Zero Project Asia Pacific Symposium 2025 with my girlfriend, Amanda. The event, a collaboration between SG Enable and the Zero Project, took place at Enabling Village, a community space here in Singapore designed for accessibility. The symposium gathered leaders in Asia to share ideas on how to remove barriers…
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Building Protection: Vaccines and Dystrophinopathy
The PPMD team was recently joined by three clinical experts, Dr. Anne Pham-Huy (Pediatric Infectious Diseases Specialist, Clinical Immunologist, and Chair of Immunize Canada), Dr. Jane Taylor (Pediatric Pulmonologist at UPMC Children’s Hospital of Pittsburgh), and Dr. Susan Matesanz (Neuromuscular Neurologist and Medical Director of Clinical In Vivo Gene Therapy at Children’s Hospital of Philadelphia)…
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In Case You Missed It…
Quest Media is an innovative adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…
