April 2026

CureDuchenne will host its annual Futures National Conference, a four-day event focused on bringing education, connection, and hope to the Duchenne and Becker muscular dystrophy (MD) community. The event will be held on May 21-24 at the JW Marriott Orlando, Grande Lakes. It will highlight the latest research and therapies for Duchenne and Becker, and feature…

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Carlee Weber and Nicole Lucas This month, the Muscular Dystrophy Association (MDA) is launching an important advocacy campaign urging Congress to advance policies that improve the lives of family caregivers in the neuromuscular community. At a time when policymakers are actively weighing changes to home and community-based services and the cost of caregiving continues to…

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This past weekend, PPMD kicked off our 2026 PPMD Together series in Philadelphia, Pennsylvania. Individuals with Duchenne and Becker, their families, clinicians, and industry partners gathered for a day and a half full of connection and community. Attendees were welcomed Friday evening with a casual adult reception to connect and reconnect with familiar faces, while…

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One problem dealing with a progressive disease like facioscapulohumeral muscular dystrophy (FSHD) is that there’s no way to predict the pace of disease progression. I think this is true for the aging process as well. In my case, it may also apply to the joint deterioration I’ve experienced following a horrifying automobile accident years ago.…

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Emerging guidance offers cautious optimism—here’s what parents and caregivers should know  For years, families affected by Duchenne muscular dystrophy have heard a consistent message: “be cautious with exercise, don’t overdo it.”  That guidance came from a place of protection. Duchenne causes muscles to break down over time, especially under strain. Without clear evidence, exercise carried unknown risks, including how much strain…

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Shalom Lim, who lives in Eastern Singapore, was diagnosed with Duchenne muscular dystrophy at 4 months old in 1996. He shares how early diagnosis shaped a lifelong approach to daily care and routines. The post Building routines that support Duchenne care appeared first on Muscular Dystrophy News.

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Edward Smith, MD, a neurologist, explains why setting clear expectations around treatment benefits and risks can help patients and families stay engaged with care plans over time. The post Setting expectations for treatment adherence in Duchenne care appeared first on Muscular Dystrophy News.

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CIAO1-related neuromuscular disorder is an inherited condition that was first reported on in late 2023. It is caused by variants in the CIAO1 gene and primarily affects muscle function, though many affected people also experience problems with the nervous system. Much about the symptoms and progression of this newly discovered condition remains to be defined.…

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The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree (vamorolone) in the European Union be expanded to include people with Duchenne muscular dystrophy (DMD) as young as 2. The CHMP is an arm of the European Medicines Agency (EMA) tasked with reviewing data on experimental therapies. The committee’s recommendation…

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On April 24, 2026, PPMD, in collaboration with the Muscular Dystrophy Association (MDA) and the Little Hercules Foundation (LHF), convened a Duchenne Newborn Screening (NBS) Stakeholder Meeting in Philadelphia. The meeting’s main goal was to gather together industry partners in Duchenne to establish a foundation of understanding of the process of state implementation, and to…

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