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I’m thankful for a big family that gives my DMD sons a full social life
The comment comes in many forms: “I don’t know how you do it.” “You have your hands full!” “I can’t imagine what it would be like.” It’s always in response to seeing my large family. I share seven children with my husband, Jason: Lexi, 24; Max, 20; Chance, 18; Rowen, 17; Charlie, 15; Mary, 11;…
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17-year-old advocate shares her NMOSD story to raise awareness
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To cope with all the changes in her life,…
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MDA 2026: ‘Body of evidence for Elevidys continues to grow’
Boys with Duchenne muscular dystrophy (DMD) who received Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial continue to show signs of slowed disease progression relative to DMD’s natural course, along with caregiver-reported improvements in everyday living. That’s according to data from the Phase 3 EMBARK clinical trial (NCT05096221) presented at the 2026…
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Sarepta Announces Plans to Submit sNDAs for AMONDYS 45 and VYONDYS 53
Today, Sarepta Therapeutics announced plans to submit supplemental New Drug Applications (sNDAs) to the U.S. Food and Drug Administration (FDA) for AMONDYS 45 and VYONDYS 53, the company’s exon-skipping therapies for the treatment of individuals living with Duchenne amenable to skipping exons 45 or 53, respectively. Both therapies are currently approved under the FDA’s accelerated…
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Sarepta Therapeutics is preparing to submit supplemental new drug applications (sNDAs) to the FDA for converting the accelerated approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals
Sarepta Therapeutics is preparing to submit supplemental new drug applications (sNDAs) to the FDA for converting the accelerated approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals, supported by data from the ESSENCE confirmatory study combined with real-world evidence. Encouragingly, the FDA has confirmed acceptance of these data for the applications, with submission planned…
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The CureDuchenne Clinic Relocates in Greater Dallas to Meet Rising Demand for Specialized Neuromuscular Care
Expanded Facility Delivers Comprehensive, Multidisciplinary Care, Removing Barriers for Patients with Duchenne and Becker Muscular Dystrophy DALLAS – February 20, 2026 – CureDuchenne, a global leader in advancing research and improving patient care for individuals with Duchenne and Becker muscular dystrophy, today celebrated the relocation and expansion of The CureDuchenne Clinic within the Neurology &…
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MDA 2026: FDA decision on deramiocel for DMD expected by August
The latest Phase 3 trial results continue showing that deramiocel, an investigational cell therapy for heart disease related to Duchenne muscular dystrophy (DMD), significantly slows the progression of arm and heart damage in boys and men with DMD. Based on the positive Phase 3 results, Capricor Therapeutics has resubmitted an application for U.S. approval of…
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Living with Duchenne grants me unique insight into accessible design
For most of my life, Duchenne muscular dystrophy has defined how I navigate the world. Duchenne is a genetic condition that causes progressive muscle loss. Over time, it weakens the arms, legs, breathing muscles, and heart. Many adults with Duchenne, including me, use wheelchairs and rely on a ventilator to breathe. Daily life often involves…
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How to find a Duchenne specialist and certified care center
Riketa Smith, a certified medical assistant, shares resources families can use to identify Duchenne specialists and certified care centers. The post How to find a Duchenne specialist and certified care center appeared first on Muscular Dystrophy News.
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Advocating for your child at Duchenne doctor visits
Sheryl Marrazzo shares advice for fellow caregivers on preparing for medical appointments, advocating with facts, and communicating effectively with clinicians. The post Advocating for your child at Duchenne doctor visits appeared first on Muscular Dystrophy News.
