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 […]
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 […]
Matches Donations to Fund Duchenne Research NEWPORT BEACH, CA (December 2, 2017) – In Week 13 of the NFL season, Clay Matthews of the Green Bay Packers will take to the field with his heart on his feet. This year, the NFL is continuing its ‘My Cause My Cleats’ campaign where NFL players design cleats […]
4th Annual Napa in Newport Wine Auction to Save the Lives of Children with Duchenne Muscular Dystrophy
Join CureDuchenne’s Celebrated Event on March 3, 2018, at the Ritz-Carlton, Laguna Niguel NEWPORT BEACH Calif., November 15, 2017 – For those who appreciate the earthen opulence of wine country, exquisite cuisine and unparalleled auction lots, Southern California’s premier wine event is back for the much-anticipated 4th Annual Napa in Newport Wine Auction, to be […]