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A virtual FDA meeting gave me the opportunity to advocate for my sons
The first thing I tell others about myself is that my husband and I share seven children: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Being a mom is literally my favorite thing about myself. Caring for my family is my main purpose in life. Most mothers will…
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Discover new science in muscular dystrophy at the 2027 MDA Conference
The next Clinical & Scientific Conference hosted by the Muscular Dystrophy Association (MDA) will take place March 21-24, 2027, in Orlando, Florida, and registration for the signature annual event — dubbed by the MDA “the leading international meeting dedicated to neuromuscular diseases” — is now open. The conference, to be held at Rosen Shingle Creek…
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Access, Impact, and Connection at the MDA Clinical & Scientific Conference
MDA National Ambassador Lily Sander at the 2026 MDA Clinical & Scientific Conference The annual MDA Clinical & Scientific Conference is the largest global gathering of leading experts dedicated to the advancement of care and research for people living with neuromuscular disease (NMD). Each year, neurologists, researchers, industry partners, and patient advocates convene to share…
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Respiratory Illness in Duchenne Muscular Dystrophy: Early Warning Signs and Care Strategies
Respiratory infections can become serious more quickly in people living with Duchenne muscular dystrophy (DMD) because respiratory muscles weaken over time. Understanding how to recognize symptoms early, use airway clearance techniques effectively, and know when to seek medical care can help reduce complications and improve outcomes. At the CureDuchenne FUTURES 2026 Annual Conference, families gathered to learn from experts about one of…
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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…
