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’s Chief Scientific Advisor, Michael Kelly, Reports From Day One of 2018 New Direction in Biology and Disease of Skeletal Muscle Conference
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 […]
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.