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

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

REGENXBIO reported new interim data from 2 additional participants in the Phase 1/2 portion of the AFFINITY DUCHENNE trial of RGX-202, an AAV8-microdystrophin gene therapy. In a 3 year old participant, microdystrophin levels at 12 weeks were 122.3% of control, and in a patient aged 7 years old microdystrophin expression was measured to be 31.5% of control. In all, RGX-202 has been well tolerated in 12 individuals, with no serious adverse events reported. In November 2024, data from the first 5 individuals treated showed functional improvements compared to natural history controls. RegenXBio indicates that we can expect to see additional Phase 1/2 data, including additional functional data, in the first half of this year. They expect to pursue FDA approval via the accelerated approval pathway in 2026. #RegenXBio #DMD #CureDuchenne #Duchenne #musculardystrophy #genetherapy #FDA https://lnkd.in/emGNb45n

CureDuchenne provided early funding to Entrada Therapeutics, who has announced that they received authorization in the United Kingdom (UK) to initiate a Phase 1/2 multiple ascending dose clinical trial in individuals with Duchenne amenable to skipping exon 45. They plan to start that trial in Q3 2025. This is the second clinical exon-skipping program for Entrada, who has authorization for exon 44 skipping trials in the UK, Europe and United States. https://lnkd.in/g_ns5-p2 #EntradaTherapeutics #exonskipping #FDA #DMD #CureDuchenne #duchenneresearch #duchenne #UK Debra Miller

Our team is back from the 2025 MDA Clinical & Scientific Conference, and we're pleased to share research updates from companies presenting on the latest advancements. From promising new therapies to groundbreaking advancements in care, we’re more determined than ever to drive progress for everyone impacted by Duchenne muscular dystrophy. Check out their news releases here:? https://lnkd.in/gfA9ZS_r #CureDuchenne #DuchenneResearch #UntilEveryoneHasACure #MDA #DMD #Duchenne

Sarepta Therapeutics has shared that a young man with Duchenne passed away following treatment with ELEVIDYS, an AAV-delivered micro-dystrophin gene therapy.?Sarepta has shared that the individual suffered from acute liver failure after treatment, and will provide more information as they further gather and analyze the data. This disease is unrelenting and new therapeutics are necessary, but can come with risks.?We are saddened for the Duchenne community, and our thoughts and prayers go out to the family and friends of this young man. https://lnkd.in/gvRJJjNt #Sarepta #DMD #Duchenne #genetherapy #cureduchenne #musculardystrophy Debra Miller

Capricor Therapeutics, Inc. received early funding from CureDuchenne, and today?Capricor?released new data reporting that individuals treated with deramiocel over three years experienced an average decline in Performance of the Upper Limb (PUL 2.0) total score of 3.46 points, compared to a 7.19-point decline in the external comparator group (p=0.019). Capricor reports that this equates to a 52% slowing of disease progression.?Capricor’s Biologics License Application (BLA) for approval for the treatment of the cardiomyopathy associated with Duchenne has been accepted by the FDA, with a with a target action date set for August 31, 2025. #cardiomyopathyphy #DMD #Duchenne #FDA #Capricor #cureduchenne #musculardystrophy #deramiocel Debra Miller https://lnkd.in/gu8eJE_2

CureDuchenne?provided early funding to?Dyne Therapeutics, and is pleased to share that Dyne has released new long-term data from the DELIVER trial in individuals amenable to skipping Exon 51.?At the 6-month time point, individuals treated with 20 mg/kg of DYNE-251 every 4 weeks had a mean absolute dystrophin expression of 8.72% of normal (adjusted for muscle content). Improvements in functional outcomes were also observed through 18 months, including improvements in Stride Velocity 95th?Centile (SV95C) that exceeded the published proposed minimal clinically important difference (MCID).?Full data from the DELIVER trial is expected in late 2025, and?Dyne plans on submitting a Biologics License Application (BLA) to the FDA for accelerated approval in early 2026. Read more: https://lnkd.in/gVGfunMT #Dyne #DMD #Duchenne #exonskipping #cureduchenne #musculardystrophy Debra Miller