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I’ve been disabled for over 50 years, and I spent over 30 years working with people with disabilities. The language surrounding disability has evolved in that time. For the most part, that’s a good thing. As an example, person-first language has become more common. It’s more appropriate to introduce someone as a person with a…

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CureDuchenne is pleased to communicate that Wave Life Sciences announced positive interim data from the ongoing Phase 2 FORWARD-53 study in Duchenne amenable skipping exon 53.  After 24 weeks of 10 mg/kg dosing every two weeks, individuals treated with WVE-N531 demonstrated an average dystrophin expression of 9.0% of normal when adjusted for muscle content (5.5%…

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As we celebrate PPMD’s Duchenne Action Month inspired by World Duchenne Awareness Day, we are filled with gratitude for the supporters who have made our work possible. We continue to celebrate PPMD’s 30th Anniversary year, a milestone that is not just about the passage of time; it’s a testament to the unwavering dedication of our…

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As an early investor in Capricor Therapeutics, CureDuchenne is happy to share that Capricor has announced their intent to apply to the FDA for full approval of Deramiocel (CAP-1002) for the treatment of Duchenne cardiomyopathy.   This BLA (Biologics License Application) filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2…

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Wave Life Sciences has announced positive interim data from the ongoing Phase 2 FORWARD-53 study of WVE-N531, an exon skipping oligonucleotide being investigated for the treatment of Duchenne in those who are amenable to exon 53 skipping. Conducted after 24 weeks of 10 mg/kg dosing every two weeks, the interim analysis indicated that WVE-N531 demonstrated…

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Capricor Therapeutics announced today its intent to file a Biologics License Application (BLA) seeking full approval of deramiocel for the treatment of Duchenne-cardiomyopathy based on existing cardiac data from their Phase 2 trials of deramiocel (CAP-1002) compared to natural history data. Deramiocel is the company’s novel cell therapy for the treatment of patients diagnosed with…

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I believe that all of us who live with a chronic illness deal with a future full of unknowns. We all wrestle with uncertainty regarding quality of life from year to year, month to month, and even day to day. My chronic illness is limb-girdle muscular dystrophy. The longer we live, the more experiences we…

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I’m going gray. Wait, I should clarify that I’ve been coloring my hair since I was 25. It started coming in gray long before I became a mom to three sons with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. But after hiding my natural color for 20 years, I’ve decided to…

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SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). The cell replacement therapy from Somite Therapeutics uses artificial intelligence (AI) and large, complex data sets to improve production of multiple human cell types. The company plans to begin clinical trials…

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We are proud to highlight the National Academies of Sciences, Engineering, and Medicine (NASEM) Consensus Study Report: Regulatory Processes for Rare Disease Drugs in the United States and European Union – Flexibilities and Collaborative Opportunities, with PPMD’s President and CEO Pat Furlong serving on the committee that helped shape its findings. This report outlines crucial…

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