March 2025

A close friend of 30 years recently asked what my life was like before I was diagnosed with limb-girdle muscular dystrophy. When I met this man, we were both in college, studying music and playing drums. It was 1991, and I’d been diagnosed with muscular dystrophy for only six years. (Originally, doctors said I had…

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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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Researchers combined advanced imaging techniques and theoretical physics to observe and explain how nanoclusters of the protein emerin form inside living cells. The study uncovers the molecular ‘rules’ driving the arrangement of emerin into nanoclusters and the mechanisms leading to their defective assembly in people with muscular dystrophy.

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In this episode of Quest Podcast, we chat with three pillars of the Duchenne muscular dystrophy community who are here with us on the 10th anniversary of PJ’s protocol. First, we have Brian Nicholoff whose son’s untimely passing was the catalyst for the creation of PJ’s protocol. Next is Amy Aikens whose son’s life was…

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The U.S. Food and Drug Administration (FDA) has accepted Capricor Therapeutics’ application seeking approval of deramiocel — a  cell therapy developed by the biotech company to treat heart muscle disease in people with Duchenne muscular dystrophy (DMD) — and granted it priority review that will speed the agency’s decision. Priority review, which shortens the review…

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Two weeks ago, I stood on stage at the Fullerton Hotel Singapore to receive a Study U.K. Alumni Award for “exceptional efforts in driving inclusion and positive change in society.” The British high commissioner to Singapore handed me the award while my partner, Amanda, and my caregiver, Glenda, cheered me on. It was a bittersweet…

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PPMD is excited to learn that the FDA has accepted Capricor Therapeutics’ Biologics License Application (BLA) for review, seeking traditional approval for deramiocel (CAP-1002). Additionally, the FDA granted the BLA Priority Review, reducing the review timeline from the standard 10-month to a priority six-month review from the date the submission is accepted by the FDA.…

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CureDuchenne Clinic within the Neuromuscular Program at CHOC Will Expand Access to Exceptional Care, Deliver Cutting-Edge Treatments, and Drive Innovative Research  Newport Beach, CA– March 3, 2025– CureDuchenne, a global leader in Duchenne muscular dystrophy (DMD) research, care, and advocacy, has announced a landmark partnership with Children’s Hospital of Orange County (CHOC) to establish the…

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Cassidy Nilles is 35 years old and lives in Illinois. Cassidy lives with Limb Girdle muscular dystrophy (LGMD) type 2J. She lives with her daughter, Capri, and their maltipoo, Mila, in a ranch-style home that they share with her parents. After spending 10 years as a cosmetologist doing hair in both Los Angeles and Illinois,…

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The Muscular Dystrophy Association (MDA) is once again hosting its annual gathering, the MDA Clinical & Scientific Conference. The 2025 event will take place on March 16-19 in Dallas at the Hilton Anatole. The four-day event will bring together scientists, clinicians, affiliated healthcare providers, academicians, advocates, and industry leaders from around the world to explore…

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