CureDuchenne

Don’t Miss This Important Update from Wave Life Sciences If you couldn’t join us live, we invite you to watch the recording of our recent webinar featuring the 48-week data update from Wave Life Sciences’ FORWARD-53 study. This insightful session offers a closer look at exon skipping therapy for Duchenne muscular dystrophy, including perspectives from Wave’s clinical and scientific leadership. Watch the full webinar here: https://lnkd.in/g97KjACm Whether you're a caregiver, clinician, or member of the Duchenne community, this is a valuable opportunity to hear directly from the experts and stay informed on progress in exon 53–skipping therapies. #exonskipping #DMD #CureDuchenne #DUCHENNE #DuchenneCommunity #webinar #FDA #wavelifesciences

THANK YOU, NASHVILLE! 💙 Even with tough weather, our CureDuchenne Cares Workshop was filled with connection, learning, and community spirit. From stretching tips to palliative care, carrier support, and the research pipeline—it was powerful to share space, meet new families, and remind everyone they’re not alone on this journey. Big thanks to our 2025 Cares Sponsors for making it all possible. Want to join us next time? See upcoming Workshops & Community Dinner Sessions: https://lnkd.in/gyEcbUDF #CureDuchenne #ThankYouNashville #DuchenneCommunity #DuchenneCare #CureDuchenneCares #DMD #DuchenneMuscularDystrophy #duchenne #musculardystrophy Avidity Biosciences, Inc. Catalyst Pharmaceuticals, Inc. Edgewise Therapeutics Entrada Therapeutics ITF Therapeutics LLC NS Pharma, Inc. PTC Therapeutics, Inc. REGENXBIO Sarepta Therapeutics

More information can be found here: https://lnkd.in/gx-wcucr

We are honoring and creating more awareness for the autism community today and every day. Let’s continue building a more unified, understanding world together. 💙 Join us at upcoming community events near you — https://lnkd.in/gyEcbUDF #WorldAutismAwarenessDay #CureDuchenne #DMD #Duchenne #duchennecommunity

Missed the Dyne Therapeutics webinar? No worries — you can watch it now! The clinical impact of the FORCE™ platform: Duchenne muscular dystrophy (DMD) An update on the DELIVER trial of DYNE-251 in males with DMD mutations amenable to exon. Watch it here: https://lnkd.in/gnGPyg5W #Duchenne #CureDuchenne #DuchenneAwareness #ClinicalTrials

Join us for a Dyne Therapeutics WEBINAR: The clinical impact of the FORCE™ platform Duchenne muscular dystrophy (DMD) – An update on the DELIVER trial of DYNE-251 in males with DMD mutations amenable to exon 51 skipping. Ashish Dugar, Ph.D., MBA Chief Medical Affairs Officer April 1, 2025 9 AM Pacific |  12 PM Eastern REGISTER: https://lnkd.in/gvNFTRvG #DMD #CureDuchenne #duchenne #webinar

Wave Life Sciences Webinar: Update on 48-week Data from FORWARD-53 Study An update on Wave's Phase 2 FORWARD-53 trial of WE-N531, Wave's exon skipping investigational therapy for Duchenne being investigated for individuals who are amenable to exon 53 skipping. We will share the recently announced 48-week FORWARD-53 data. REGISTER TODAY! https://lnkd.in/gteA9wQj #FDA #Duchenne #DMD #RESEARCH #CureDuchenne

Looking forward to an inspiring exchange of ideas on “Ensuring Global Access to Technology" with an accomplished group of leaders at the Longwood Healthcare Leaders Longwood Fund Miami CEO conference on April 10. I’m passionate about the power of venture philanthropy to accelerate innovation and deliver life-changing treatments to those who need them —especially those affected by Duchenne muscular dystrophy. I look forward to sharing our experience at CureDuchenne building a model that not only fuels scientific breakthroughs, but ensures those breakthroughs reach the families who need them most. Learn more and register: https://lnkd.in/gmAfsrsy #longwoodhealthcareleaders #MiamiCEO #venturephilanthropy #healthcareinnovation #CureDuchenne

Wave Life Sciences has shared new data from its ongoing FORWARD-53 clinical trial, evaluating the investigational exon-skipping therapy WVE-N531 for individuals with Duchenne muscular dystrophy amenable to exon 53 skipping. Key findings after 48 weeks of treatment: -Dystrophin expression remained stable between weeks 24 and 48, with an average of 7.8% dystrophin observed and 88% of boys exceeding 5%. -Time-to-Rise improved by an average of 3.8 seconds compared to natural history, a statistically and clinically significant difference. - Muscle biopsy analysis showed a reduction in fibrosis, inflammation, and muscle necrosis, as well as a shift toward more mature, healthier muscle tissue. Wave Life Sciences plans to pursue FDA accelerated approval in 2026 and expand development to other exon targets based on this data. Read the community letter and press release: https://lnkd.in/gqC4tJXY #Duchenne #ClinicalTrials #CureDuchenne #ResearchUpdate #DMD #exonskipping

Wave Life Sciences has shared new data from its ongoing FORWARD-53 clinical trial, evaluating the investigational exon-skipping therapy WVE-N531 for individuals with Duchenne muscular dystrophy amenable to exon 53 skipping. Key findings after 48 weeks of treatment: -Dystrophin expression remained stable between weeks 24 and 48, with an average of 7.8% dystrophin observed and 88% of boys exceeding 5%. -Time-to-Rise improved by an average of 3.8 seconds compared to natural history, a statistically and clinically significant difference. - Muscle biopsy analysis showed a reduction in fibrosis, inflammation, and muscle necrosis, as well as a shift toward more mature, healthier muscle tissue. Wave Life Sciences plans to pursue FDA accelerated approval in 2026 and expand development to other exon targets based on this data. Read the community letter and press release: https://lnkd.in/gqC4tJXY #Duchenne #ClinicalTrials #CureDuchenne #ResearchUpdate #DMD #exonskipping