CureDuchenne

?? Today, on Rare Disease Day, we stand in solidarity 300 million people worldwide living with a rare disease. At CureDuchenne, we are relentlessly driving research forward, investing in innovative therapies, and supporting families every step of the way. Our mission is clear: to find a cure and improve the lives of those impacted by Duchenne muscular dystrophy. Together, we will cure Duchenne. ?? What You Can Do Today: ?? Learn about Duchenne and share this post to spread awareness.??? Advocate for disease research.??? Donate to fund life-saving research and care programs. Visit our website https://cureduchenne.org #RareDiseaseDay #CureDuchenne #UntilEveryoneHasACure ?

CureDuchenne was an early funder of Avidity, and today we share an important milestone: Enrollment is now complete for the EXPLORE44-OLE clinical trial. Avidity Biosciences, Inc. is pursuing accelerated approval for del-zota in the U.S., with plans to file a Biologics License Application (BLA) by the end of the year. Stay tuned—top-line data from the EXPLORE44? Phase 1/2 trial will be shared next month. #Exon44 #Exonskipping #CureDuchenne #DuchenneResearch #UntilEveryoneHasACure #DMD #Duchenne #MuscularDystrophy #FDA #research Learn more: https://lnkd.in/g2cGTwGA

Bid for a Cure. Bid for Their Dreams. At Napa in Newport, our live and silent auctions are more than just bids—they are lifelines for boys fighting Duchenne muscular dystrophy. Every paddle raised and every silent auction win brings us closer to life-changing treatments and, ultimately, a cure. Meet the boys who inspire us: - Cooper finds his voice through music, writing and performing songs that move audiences. - Hunter is telling his story through his own superhero comic book, inspiring kids like him to chase their dreams. - Thomas dreams of becoming a doctor and brings his imagination to life through art. - Turbo has an unstoppable love for speed, dreaming of visiting the Porsche Museum in Germany. - Dillon has a passion for exploring the world, immersing himself in cultures and history. - Henry dreams of becoming a Disney Pixar animator, creating magic for the world to enjoy. ?? Your bid fuels their dreams. Your generosity accelerates groundbreaking Duchenne research, helping these boys live longer, fuller lives. To bid by proxy in the live auction, email [email protected] To participate in the silent auction, click here: https://lnkd.in/gxdTQfsY Let’s raise our paddles, open our hearts, and make a difference March 1, 2025, at Pendry Hotels & Resorts in Newport Beach. Tickets: https://napainnewport.org/ Together, we can turn dreams into reality. #NapaInNewport #BidForACure #CureDuchenne #WineForACause #Philanthropy #Duchenne #FundACure

Mack, Jack & McConaughey announced the #MJM2025 lineup for the Jack & Friends singer-songwriter event on Friday, April 25th! Get ready for an unforgettable night with performances by Jack Ingram, Kaitlin Butts,?Natalie Hemby?& More! Get your tickets here: https://lnkd.in/gf5Jr7Hk?

As an early funder of Entrada Therapeutics, we are pleased to share that the FDA has removed the clinical hold on ENTR-601-44, the experimental therapeutic designed for individuals with Duchenne amenable to skipping exon 44. ENTR-601-44 is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a proprietary Endosomal Escape Vehicle (EEV?), which aims to facilitate delivery of the exon-skipping PMO to the right compartment within the muscle. Entrada will conduct the ELEVATE-44-102 trial in the US, a Phase 1b multiple ascending dose study in 32 ambulatory and non-ambulatory adults with Duchenne. Dosing will be every 6 weeks, with 4 different dosing levels planned. Entrada will be looking at safety, tolerability, and target engagement as measured by exon skipping and dystrophin production. Entrada also plans to conduct a global, two-part, randomized double-blind placebo controlled Phase 1/2 study, ELEVATE-44-201, outside of the US.

What an incredible day at the Invest in Cures conference! We were honored to have our CEO and Founder, Debra Miller, represent CureDuchenne on a distinguished panel discussing how venture philanthropy accelerates life-changing treatments. Our strategic investment model has helped advance 18 potential therapies into human clinical trials to push groundbreaking research forward. Thank you to LaunchBio Inc. and Rebecca Beattie for hosting such an impactful and inspiring event. It was a privilege for Debra to share the stage with Karyn DeMartini, MBA, Eunice Chapon, and moderator Lydia McClure as they explored the future of mission-driven investment in healthcare. We’re excited for what’s ahead as we continue to turn innovation into impact! #InvestInCures #VenturePhilanthropy #CureDuchenne #DuchenneResearch #ImpactInvestment #DMD #MD

I can’t wait for this year FUTURES National Conference!

Meet the panel! Investment Philosophies of Leading Venture Philanthropy Funds will feature: ? Karyn DeMartini, MBA, March of Dimes Innovation Fund? ? Debra Miller, CureDuchenne ? Eunice Chapon, BrightEdge ?? February 20 ?? San Diego ?? Learn more and register now: https://lnkd.in/e4J-5ccZ Together with UC San Diego, Curebound and Fondation Ipsen along with sponsor BioLabs and VIP dinner sponsor Wilson Sonsini Goodrich & Rosati. #InvestInCures #Oncology #RareDiseases #Biotech #LifeSciences #CancerResearch #LaunchBio #BuildingBetterBiotechs

It's not too late to register for this forum featuring our own Debra Miller and other leaders in rare disease research, biotech innovation, and venture philanthropy to discuss how smart investments can drive life-changing breakthroughs. Learn more and register: https://lnkd.in/eypKstag

Solid Biosciences, announced positive initial results from INSPIRE DUCHENNE, their next-generation gene therapy clinical study of SGT-003.?Interim 90-day biopsy data reported in the first three participants showed an average microdystrophin expression of 110%, as measured by western blot, and improvements in multiple biomarkers that are indicators of muscle health and resilience.?SGT-003 has been well-tolerated across the six patients dosed to date. No?serious adverse events (SAEs) were observed.?There are currently six active clinical trial sites across the United States and Canada, and they have regulatory approvals to expand to the U.K. and Italy. In mid-2025, the Company plans to request a meeting with the FDA to discuss the potential accelerated approval pathway for SGT-003. Read more in the link: #Duchenne #DMD #research #FDA #CureDuchenne