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This August will mark 40 years since I was diagnosed with what we eventually discovered was limb-girdle muscular dystrophy. Though it’s taken me many years to accept my condition, it’s progressed slowly over the past four decades — and in that way, it’s somewhat akin to the movement in the United States that’s focused on…

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Yesterday, we received news that the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) has been terminated, effective immediately. For over 20 years, this committee has served as the guiding body for adding conditions to the Recommended Uniform Screening Panel (RUSP)—a critical step in ensuring that newborns across the country have access to…

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I don’t gamble. I don’t know how to do it. I wouldn’t even know how to place a bet, and the only gambling game I recognize by name is poker. However, for someone who’s never gambled, I sure understand what it must feel like. How? Because I’m the caregiver of three young men living with…

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KER-065, a potential treatment for Duchenne muscular dystrophy (DMD) and other neuromuscular diseases, showed a good safety profile in a Phase 1 clinical trial. No serious side effects were seen in the trial, which tested single and multiple doses of KER-065 in healthy volunteers, and no major safety issues were noted. Data also indicate that…

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Craig Helget and his brother, Terry Helget, at MDA Summer Camp in 1985. The Helget family has been volunteering at MDA Summer Camp for more than four decades. It all started with Craig, who lived with Duchenne muscular dystrophy, attending camp for the first time in 1984. Nine-year-old Craig returned home from his first week…

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This past weekend, PPMD was joined by families, individuals with Duchenne and Becker, clinicians, researchers, industry partners and local vendors for 2 days of community building, knowledge sharing, and meaningful connections at our PPMD Together event in Minneapolis, MN.  The event started with a social gathering on Friday evening for parents, caregivers and adults with…

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In this Quest Podcast episode, we chat with MDA Ambassador Jess Westman about embracing individuality and finding joy in our lives as paths and priorities change. The activist, actor, composer, author, and podcaster has devoted his career to providing joy and laughter to others and finds personal fulfillment through his faith and advocacy. Jess joins…

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Avidity Biosciences recently joined PPMD for a community webinar to share topline data from their EXPLORE44® clinical trial, which is assessing the safety and efficacy of the investigational therapy delpacibart zotadirsen (formerly AOC 1044, abbreviated as del-zota) in people living with Duchenne who are amenable to exon 44 skipping. The Avidity team shared background information…

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The use of experimental gene therapy BB-301 led to improvements in swallowing ability for the first three people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakening in the throat and eyes — in a clinical trial. That’s according to interim data shared at the Muscular Dystrophy Association‘s 2025…

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The neuromuscular junction (NMJ) disorders are a group of conditions that disrupt the communication between motor neurons and muscles, resulting in muscle weakness, fatigue, problems with movement and mobility, and, in severe cases, paralysis. Recognized NMJ disorders include the autoimmune disorders myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS), the genetic conditions known as congenital myasthenic…

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