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FDA Accepts sNDAs to Review Traditional Approval for AMONDYS 45 and VYONDYS 53
Today, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Applications (sNDAs) seeking traditional approval for AMONDYS 45® (casimersen) and VYONDYS 53® (golodirsen). The FDA will now begin its review, and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of February 28,…
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FDA accepts Sarepta’s application to convert AMONDYS 45 and VYONDYS 53 from accelerated to full approval
Proposed text: CureDuchenne was an early funder of Sarepta Therapeutics, and we are pleased to share that the US FDA has accepted supplemental New Drug Applications (sNDAs) for AMONDYS 45 and VYONDYS 53, for individuals with Duchenne amenable to skipping exon 45 and exon 53, respectively. The FDA is targeting a PDUFA decision date of…
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July meeting date set for FDA, developer to talk deramiocel for DMD
Amid its ongoing review of the cell therapy deramiocel for people with Duchenne muscular dystrophy (DMD), the U.S. Food and Drug Administration (FDA) has scheduled a meeting with Capricor Therapeutics, its developer, to discuss the company’s resubmission of an application seeking the treatment’s approval. That meeting — between Capricor and the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee, or…
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Protecting mobility in Duchenne starts early
Brenda Song, MD, is a neurologist who directs the UMass Duchenne Muscular Dystrophy Program, and she is a pioneer in organizing multidisciplinary care for Duchenne populations. She explains how physical therapy, stretching, and supportive equipment can help people with Duchenne maintain mobility and manage contractures. The post Protecting mobility in Duchenne starts early appeared first…
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Taking advantage of charting software to track Duchenne
Sheryl Marrazzo explains why tracking baseline measurements, medications, symptoms, and health trends has become an important part of managing her son’s Duchenne muscular dystrophy and advocating for his care. The post Taking advantage of charting software to track Duchenne appeared first on Muscular Dystrophy News.
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Simply Stated: Updates in Myofibrillar Myopathies (MFMs)
Myofibrillar myopathies (MFM) are a group of inherited muscle disorders characterized by progressive muscle weakness and the abnormal accumulation of proteins within muscle cells. These disorders have been linked to variants in numerous genes and can present with a wide range of symptoms and severity. MFMs are considered rare diseases, and their exact prevalence remains…
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Catching Up on PPMD’s 2026 Annual Conference: Part II
PPMD’s 2026 Annual Conference brought together individuals and families from across the Duchenne and Becker community for three days filled with scientific presentations, discussions around care, clinical trials and quality of life, and of course, social events to connect and recharge. To all of our attendees, speakers, and sponsors, THANK YOU! Brain & behavior The…
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MDA’s Guide to the Affordable Care Act (ACA)
For many people living with neuromuscular diseases, access to reliable health insurance can be the difference between receiving life-changing care and going without it. The Affordable Care Act (ACA), signed into law in 2010, created stronger protections for people in the United States living with disabilities, chronic illnesses, and preexisting conditions. Understanding the ACA can…
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An annual physical brings a sigh of relief to this Duchenne MD caregiver
I had my annual physical last week and when the doctor told me I was in good health, it was a relief. I let out a breath I hadn’t realized I was holding. I am a mother to seven: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max,…
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FDA Announces Advisory Committee Meeting to Review Capricor Therapeutics’ Biologics License Application for Deramiocel
The U.S. Food and Drug Administration (FDA) has scheduled an Advisory Committee meeting for July 29, 2026, to review Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy. The application remains under review with a PDUFA target action date of August 22, 2026. Advisory Committee…
