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 […]
CureDuchenne is proud that another one of our funding projects is entering clinical trial. Pfizer Inc. is planning a Phase 1b, Multicenter, Open-label, Single Ascending Dose Study to Evaluate the […]
Today, November 28, join CureDuchenne for #GivingTuesday, the global giving day that connects millions of people together to support and champion the causes they believe in by donating and fundraising on […]
When Thanksgiving comes around each year, I’m reminded of how much we actually have to be thankful for. As a Duchenne parent, I am thankful every day for the moments […]
Dr. Eric Olson to Provide CRISPR/Cas9 and Gene Therapy Update During November 16 Webinar We are delighted that Exonics Therapeutics has closed a $40 million Series A financing with […]
