Press Releases

NEWPORT BEACH, Calif. – (BUSINESS WIRE) – CureDuchenne, a national venture philanthropy nonprofit focused on finding a cure for Duchenne muscular dystrophy (DMD), announced today a $500,000 investment into 4D Molecular Therapeutics (4DMT), a world-leader in Therapeutic Vector Evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development, in an effort to advance […]
NEWPORT BEACH, Calif.–(BUSINESS WIRE)–The DuchenneXchange.org has a reason to celebrate on World Duchenne Day. The dedicated online platform for the Duchenne muscular dystrophy (DMD) community announced today it has surpassed enrollment goals of partner organizations and users since it was introduced to the community just five weeks ago. To date, 13 Duchenne-related nonprofit organizations and 700 community […]
Dear Friends, Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in dystrophin restoration of 92% in the heart of the canine’s. As the only Duchenne nonprofit organization to support Dr. Eric Olson’s work through Exonics, and […]
NEWPORT BEACH, CALIFORNIA, AUGUST 17, 2018 – CureDuchenne, a leading nonprofit focused on funding research to find a cure for Duchenne muscular dystrophy, is thrilled and honored to announce that ActivePDF will serve as the Presenting Sponsor for the 8th Annual Getzlaf Golf Shootout for the 3rd year in a row. “This is more than […]
DuchenneXchange, the dedicated online platform built by the Duchenne community for the Duchenne community, will host a free hour-long webinar for Duchenne community members to learn about the benefits of the DuchenneXchange on Tuesday, August 7, 2018 at 1:00 PM (PDT) / 4:00PM (EDT). Those interested are encouraged to register and dial-in at: Registration Link […]
Dedicated Online Platform DuchenneXchange.org Launches to Unite, Support and Educate Duchenne Community Collaborative site offers a trustworthy, moderated online space where Duchenne families, caregivers, NGOs and pharmaceutical companies can connect and share knowledge NORWALK, CT (July 31, 2018) — The Duchenne muscular dystrophy (DMD) community now has a dedicated online platform where they can connect […]
PTC Therapeutics Update on Ataluren Study 041 PTC Therapeutics is enrolling boys and young men with a nonsense mutation in a clinical trial for Ataluren.  To ensure that participants who enroll in Study 041 get the most benefit, and to ensure their safety, there are strict criteria around who is able to enter the study. […]
The third day of the conference focused on stem cells, new small molecule screens, poster sessions and clinical trial updates. Hirofumi Komaki (National Center of Neurology and Psychiatry, Japan) presented results of a Japanese Phase I/II dose-finding clinical study with NS-065, an exon-53 skipping drug (NS Pharma) for the treatment of Duchenne. The drug was […]
Today, Pfizer announced that the first patient was dosed in their mini-dystrophin gene therapy trial.  This is an exciting day for the Duchenne community.  Pfizer has the experience to conduct this trial, and manufacturing capacity to see it through to success.  Of course, this is an early stage safety trial and there is a long […]
Monday, I attended a meeting in Washington DC, hosted by Duke University’s Center for Health Policy, which included three sessions: Using Prior Data from Early Phase Trials to Inform Phase 3 Designs Utilizing Patient Registry and Natural History Study Data to Advance Therapeutic Development for Rare Diseases Leveraging Master Protocols for Trials with Small Patient […]
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