September 2024

The Rev It Up 5K presented by Mitsubishi Electric Automotive America held their 4th annual event on September 21, 2024, bringing in $27,000 for PPMD! Thank you to Jason Dempsey for organizing this event and making it incredibly fun and memorable for so many. It would not have been the success that it was without…

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As part of our ongoing Cardiac Initiative, PPMD is proud to host the upcoming professional meeting, “Expanding Access to Heart Transplantation: Focus on Muscular Dystrophy,” taking place September 27-28, 2024, at Stanford University. This important event will bring together leading experts in cardiology, neuromuscular disorders, and transplant medicine to address a critical but often overlooked…

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Capricor Therapeutics will soon begin filing an application seeking U.S. approval of its cell therapy deramiocel for cardiomyopathy, a disease of the heart’s muscle, in people with Duchenne muscular dystrophy (DMD). Following recent meetings with the U.S. Food and Drug Administration (FDA), Capricor will file a rolling application, meaning the company can submit parts of…

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Parent Project Muscular Dystrophy (PPMD) is pleased to announce our participation in the upcoming “Respiratory Assessment and Management of Duchenne Muscular Dystrophy: Current Standards and Future Directions” meeting, which will take place in Rome, Italy, from September 26–27, 2024. This important event will bring together experts from around the world to discuss advancements in respiratory…

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The Muscular Dystrophy Association (MDA) is providing neuromuscular disease (NMD) patients and their families with the opportunity to meet experts and other people on similar journeys through its Engage Symposiums, free in-person events that include disease-specific sessions as well as talks focusing on NMDs in general. The association is offering several symposiums this year, with…

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This past weekend, PPMD convened over 100 attendees at our PPMD Together event in Seattle, Washington. Families, individuals with Duchenne and Becker, clinicians, researchers, industry partners and local vendors came together for the 2 day event to connect, share experiences and learn from each other. The event started with a social gathering on Friday evening…

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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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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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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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