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Thank You for Raising Your Voices During the Deramiocel FDA Advisory Committee Meeting
Today, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) met to discuss Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel as a potential treatment for Duchenne muscular dystrophy (Duchenne). Following a full day of presentations, discussion, and public testimony from patients, families and physicians, the committee voted 3 Yes – 9 No on…
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How respite care gives my family caregivers much-needed travel breaks
I returned home this past Saturday from my first time in a respite care facility, where I’d been staying for nine days. The backstory is that my wife, Wendy, and daughter, Jill, who are my primary caregivers, love to travel together. In the past, I’ve been able to manage daily living while they were away…
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Exercise and Physical Therapy in Duchenne Muscular Dystrophy: What Families Need to Know
When people think about physical therapy with Duchenne muscular dystrophy, they often picture stretching exercises or clinic visits. But at the CureDuchenne FUTURES 2026 Annual Conference, physical therapists Doug Levine, PT, and Jennifer Wallace, PT , reminded families that movement and mobility encompass much more than muscles and joints. From equipment and exercise to school accommodations and daily routines, every aspect…
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Navigating Combination Therapies in Duchenne Muscular Dystrophy
Just a few years ago, families attending CureDuchenne’s FUTURES 2026 Annual Conference were weighing whether to participate in clinical trials. Today, the conversation has shifted dramatically. With multiple approved therapies now available for Duchenne muscular dystrophy (DMD), families and clinicians face a new question: how should these treatments be used together? During a FUTURES panel discussion moderated by CureDuchenne Chief Medical Advisor Brenda Wong,…
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Ahead of the July 29 Advisory Committee: We Urge the FDA to Consider the Totality of Evidence
On July 29th, 2026, the FDA will convene an Advisory Committee meeting to evaluate Deramiocel, Capricor Therapeutics’ investigational therapy for Duchenne muscular dystrophy. These meetings are an important step in the regulatory process, bringing together independent experts, community voices, and scientific data to inform FDA’s review. For our community, this moment represents more than a…
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Mental Health, Independence, and Transition to Adulthood in Duchenne Muscular Dystrophy
As children with Duchenne muscular dystrophy grow, families often find themselves navigating new questions that extend beyond medical care. How can parents encourage independence while still providing support? What resources can help prepare for adulthood? And how do mental health and emotional well-being fit into the journey? At the CureDuchenne FUTURES 2026 Annual Conference, social workers Kayla Richards and Ruchi Patel, CureDuchenne Family…
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Support from Duchenne caregivers must include boundaries
Hawken Miller, who lives in Newport Beach, California, was diagnosed with Duchenne muscular dystrophy at age 5 in 2002. He shares why caregivers should support, rather than take over, when helping someone follow a treatment plan, and why building independence can help patients develop confidence in managing their own care. The post Support from Duchenne…
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Why judgment-free Duchenne noncompliance conversations matter
Edward Smith, MD, a neurologist, explains why he avoids judgmental approaches to noncompliance and instead focuses on trust, understanding barriers to care, and collaborative decision-making with patients and families. The post Why judgment-free Duchenne noncompliance conversations matter appeared first on Muscular Dystrophy News.
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Continued steroid use tied to slower lung decline in adults with DMD
Continuing corticosteroid treatment into adulthood was associated with slower lung-function decline in people with Duchenne muscular dystrophy (DMD), according to a new study. “The results of this study provide further evidence to support continuing [corticosteroid] treatment into late adolescence and adulthood” for people with DMD, the researchers wrote. The study, “The Effect of Corticosteroids on…
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What to Know Ahead of This Week’s Deramiocel FDA Advisory Committee Meeting
On Wednesday, July 29, 2026, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) will meet to review Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel as a potential treatment for Duchenne. The meeting marks an important step in the FDA’s review process and provides an opportunity for independent experts to discuss the available…
