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Exon 44 Skipping CureDuchenne was an early funder of Avidity Biosciences, which is now a Novartis company.  We are pleased to share that they have submitted a BLA to the FDA for delpacibart zotadirsen (“del-zota”) as a treatment option for individuals with Duchenne amenable to skipping exon 44.  This application was submitted through the FDA’s…

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REGENXBIO has completed dosing in the confirmatory study of RGX-202, an AAV-delivered microdystrophin gene therapy for the treatment of Duchenne.  The company is on track to initiate a Biologics License Application (BLA) under the accelerated approval pathway in Q3 2026, for a potential approval by the US FDA in the second half on 2027. Press…

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Hello! In this blog, I’m sharing accessible beauty and fashion tips I’ve collected through plenty of trial and error. Beauty and fashion are major forms of self-expression for me, and I know I’m not alone in that. People with disabilities deserve to present themselves to the world in whatever way feels authentic and comfortable to…

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The CITGO Lake Charles Refinery raised more than $750,000 to support the Muscular Dystrophy Association (MDA) at its 41st annual MDA Golf Classic, an annual golf fundraiser. Since 1985, CITGO Lake Charles has raised $8.5 million for the MDA. The total amount raised this year — $756,800 — set a new record. As in past…

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Some events are hard to picture before you attend. You may wonder who will be there, whether the topics will apply to you, or if it is worth carving out the time. MDA Engage is designed for people living with neuromuscular disease, families, caregivers, and loved ones who want practical information, real conversation, and a…

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Each year, I attend at least one national gathering of people living with muscular dystrophy. In addition to learning about the most updated care standards, treatment strategies, and research initiatives, I’m able to bond with those who share a similar diagnosis, and have made some dear friends. The upcoming Parent Project Muscular Dystrophy (PPMD) 2026…

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On June 22, 2026, PPMD Chief Executive Officer  Katherine Beaverson, MS attended the U.S. Department of Health and Human Services (HHS) Clinical Trials Roundtable in Washington, D.C., joining leaders from across government, industry, academia, and the patient advocacy community.  The roundtable, convened by HHS leadership, focused on a central challenge: how to cut the duration…

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June is a quiet month here in rural Nebraska. Most days, only neighbors drive on the street outside our home, and you can hear the wind moving through the plains. That may sound boring to some of you from the big cities, but here it is supposed to be relaxing and serene. My children have…

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Rob Roozeboom is the founder of RISE Ministries and the creator of RiseFest, one of the largest Christian music festivals in the Midwest. Living in Iowa with muscular dystrophy, Rob is passionate about sharing hope, encouraging others through faith, and advocating for people living with disabilities. He and his wife are proud parents who believe…

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Health Canada has accepted and granted priority review to Italfarmaco’s application seeking the approval of oral givinostat, sold in the U.S. under the brand name Duvyzat, to treat Duchenne muscular dystrophy (DMD). The priority review status is reserved for therapies that could significantly improve the benefit-risk profile over current treatments. This designation shortens the evaluation…

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