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 […]
NEWPORT BEACH, Calif., March 21, 2018 – Hundreds of people gathered at the Ritz-Carlton on Saturday, March 3 to enjoy the best of Napa Valley wineries and help find a […]
Today we announced that the U.S. Food and Drug Administration has placed our Phase I/II clinical trial for SGT-001, IGNITE DMD, on Clinical Hold following a serious adverse event that occurred in the first patient dosed, a non-ambulatory adolescent. The patient was admitted to the hospital, received treatment and, as of the writing of this letter, is home with his family with no symptoms. Details about the event can be found in the press release we issued today, which is available here. The team at Solid will be working with the principal investigator and FDA to fully understand the cause and nature of this event, as well as identify appropriate next steps as soon as possible.
Dr. Wagner and the CureDuchenne team provided information about Santhera Pharmaceuticals’ expanded access and compassionate use in the U.S., the BreatheDMD program, and Santhera answered questions from the community.
NEWPORT BEACH, Calif., Feb. 2, 2018 /PRNewswire/ — CureDuchenne, a nonprofit that funds research to find a cure for Duchenne muscular dystrophy, pioneered the community-based CureDuchenne Cares program which provides families, caregivers, and individuals living […]
We are sharing a holiday message and community update from PTC Therapeutics. Dear Duchenne Community, As we enter the holiday season, I wanted to thank the community for all your […]
