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 […]
Dear Friends, We are pleased to provide you with detailed information from Santhera Pharmaceuticals about an ongoing clinical trial that your son may be able to participate in. Beyond investing in early scientific research to accelerate a pathway to a cure for Duchenne, CureDuchenne is committed to providing the Duchenne community with education, help and […]
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 […]
The second day of the conference focused on gene editing, gene therapy as well as the latest research into the development of disease biomarkers. Presentations included: Dongsheng Duan (University of Missouri) described his research groups efforts that led to the selection of an optimized micro dystrophin gene construct for systemic AAV deliver to Duchenne patients. […]
CureDuchenne staff is participating in the New Direction in Biology and Disease of Skeletal Muscle Conference in New Orleans this week. Our staff scientist, Michael Kelly, will be providing a summary of key learnings each day. Have a question? Leave us a comment and Michael will respond. Jane Owens (Rare Disease Research Unit, Pfizer) provided an […]
This was one of those mornings the Duchenne community will remember! Sarepta announced that the biopsies taken from the first three gene therapy patients showed, on average, over 38% dystrophin, as measured by western blot. This is a significant amount of dystrophin, and could be a game changer for Duchenne. Even though this is early […]
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 […]