Uncategorized

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…

Know More

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…

Know More

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…

Know More

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%…

Know More

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…

Know More

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…

Know More

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…

Know More

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…

Know More

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…

Know More

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…

Know More