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 […]
Dr. Gilmore O’Neill, Chief Medical Officer of Sarepta Therapeutics, Inc, gave a scientific overview of Sarepta’s RNA-targeted exon skipping technologies, PMO and PPMO. Dr. O’Neill provided valuable information on this […]
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 […]
CureDuchenne hosted a webinar with Dr. Paula Clemens and Lauren Morgenroth on the NS Pharma Exon 53 Skipping Program. The webinar was an opportunity for the Duchenne community to learn […]
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 […]
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 […]
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 […]
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 […]
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 […]
