August 2026

ASK ME ANYTHING: Endocrine & Bone Health for People with DBMD Dr. David Weber, Pediatric Endocrinologist at Children’s Hospital of Philadelphia (CHOP) Recorded: Wednesday, August 5, 2026 Watch HERE The post ASK ME ANYTHING: Endocrine & Bone Health for People DBMD appeared first on CureDuchenne.

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Watch our webinar (in English and Spanish) with helpful insights on who is eligible, common school supports, transition planning, and how to build a strong partnership with your child’s school. Recorded: August 2026Watch HERE The post ASK ME ANYTHING: Navigating IEPs and 504 Plans appeared first on CureDuchenne.

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“I don’t even know what question to ask.” I’ve heard some version of that sentence hundreds of times. Sometimes it’s from a parent whose child was just diagnosed with Duchenne muscular dystrophy. Sometimes it’s a husband whose wife has been diagnosed with ALS. Other times it’s someone living with spinal muscular atrophy, Charcot-Marie-Tooth disease, limb-girdle…

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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…

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Shortly after being diagnosed with muscular dystrophy in 1985, I returned to the hospital for a checkup and to review my treatment plan. I thought the doctors might tell me to eat an apple, take some aspirin, and rest up. I was 12 years old, so what did I know? In my mind, I’d go…

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The University of Alabama at Birmingham (UAB) has launched an expanded access program to make the experimental exon-skipping therapy delpacibart zotadirsen (del-zota) available to qualifying patients with Duchenne muscular dystrophy (DMD). Del-zota, which is being developed by Avidity Biosciences, is specifically designed to treat DMD in people with disease-causing mutations that are amenable to exon…

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The Civil Rights Movement of the 1960s prompted the passage of several landmark civil rights laws, including the Fair Housing Act (FHA), which was enacted in 1968 to protect people from discrimination when renting, buying, or financing a home. What is the FHA? “From a broad perspective, the FHA is about preventing discriminatory treatment for…

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An advisory committee to the U.S. Food and Drug Administration (FDA) has voted that available evidence does not support the effectiveness of the experimental cell therapy deramiocel for treating heart muscle disease in people with Duchenne muscular dystrophy (DMD). In a company press release, Capricor Therapeutics, the therapy’s developer, said the question before the committee…

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In this episode of the Quest Podcast, we chat with Cerys Davage, a Welsh podcaster and content creator living with limb-girdle muscular dystrophy (LGMD), who has turned her diagnosis into a platform for connection and representation. Cerys opens up about growing up in a family that chose openness over silence, the moment in university when…

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CureDuchenne has submitted comments to the U.S. Food and Drug Administration (FDA) on several proposed policies that could affect how new gene therapies and other genetic treatments are developed and reviewed for Duchenne muscular dystrophy, Becker muscular dystrophy, and other rare diseases.  As a nonprofit organization focused on improving lives of people with Duchenne, CureDuchenne works with patients, families, researchers, doctors, companies, and regulators to help bring promising therapies to people who need them as quickly and safely as possible.  Why These FDA Policies Matter for Families …

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