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 […]
Napa In Newport Raises More Than One Million Dollars To Help Find a Cure for Duchenne Muscular Dystrophy
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 cure for Duchenne muscular dystrophy. The fourth annual Napa in Newport wine auction, powered by Karma, raised more than one million dollars to support CureDuchenne’s […]
Letter to the Duchenne community from Solid Biosciences about the status of the IGNITE DMD Clinical Trial
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.
CureDuchenne Cares Begins 6th Season of Family Workshops and Summit Series in Oklahoma City, Oklahoma
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 with Duchenne an interactive education and supportive program. With support from biotech and pharmaceutical companies, CureDuchenne Cares brings to local communities critical resources that can positively […]
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 support throughout the regulatory process for Translarna™ (ataluren) with the FDA. Your voice as passionate advocates has been instrumental in this process. As you know, […]
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 Safety and Tolerability of PF-06939926 in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD). This clinical research study is designed to evaluate the safety and tolerability […]
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 their behalf. Following the Thanksgiving holiday and the kickoff of the holiday shopping season, #GivingTuesday is meant to inspire people to give back to charities […]