October 2025

I’ve always had a robust sense of humor. Some people have told me that I’m witty and quick to respond to a funny situation while also defusing tense conversations with laughter. I think it’s a piece of my armor that protects me from my rare chronic illness, limb-girdle muscular dystrophy. (That’s type 2E/R4 for those…

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A new threshold study, “Re-evaluating Acceptable Risk of Death from Gene Therapy,” has been published in the European Journal of Medical Genetics. This work addresses a critical question: how should risk and benefit be assessed and balanced in potentially life-changing gene therapies for conditions like Duchenne muscular dystrophy? In developing and considering therapeutic options, individuals…

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Faith is 14 years old and lives in Texas. She is a freshman in high school. Faith served as the MDA National Ambassador in 2018 and 2019. She is currently serving as her Freshman Class President. Faith loves the time she has spent at MDA Summer Camp, and in her spare time she loves to…

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Brogidirsen (NS-089/NCNP-02), NS Pharma’s investigational therapy for people with Duchenne muscular dystrophy (DMD) who are amenable to exon 44 skipping, has been shown to sustain motor function and maintain a favorable safety profile over 3.5 years of treatment in an open-label extension study. The therapy is designed to restore the production of a functional version…

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In the past couple months, I’ve had more medical challenges than usual. My right arm hasn’t been working well, leading to some additional struggles with activities of daily living. In June, what I thought was a bug bite appeared on the knuckle of my right middle finger. It would swell up then burst open every…

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Scientists discovered that lean pork builds muscle more effectively post-workout than high-fat pork, even with identical protein levels. Using advanced tracking techniques, they found that fat content blunted the body’s muscle-building response. The results contradict previous findings about fattier foods enhancing synthesis, suggesting that food form and processing matter.

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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, 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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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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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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