NEWPORT BEACH, CALIFORNIA, AUGUST 17, 2018 – CureDuchenne, a leading nonprofit focused on funding research to find a cure for Duchenne muscular dystrophy, is thrilled and honored to announce that ActivePDF will serve as the Presenting Sponsor for the 8th Annual Getzlaf Golf Shootout for the 3rd year in a row. “This is more than […]
DuchenneXchange, the dedicated online platform built by the Duchenne community for the Duchenne community, will host a free hour-long webinar for Duchenne community members to learn about the benefits of the DuchenneXchange on Tuesday, August 7, 2018 at 1:00 PM (PDT) / 4:00PM (EDT). Those interested are encouraged to register and dial-in at: Registration Link […]
Dedicated Online Platform DuchenneXchange.org Launches to Unite, Support and Educate Duchenne Community Collaborative site offers a trustworthy, moderated online space where Duchenne families, caregivers, NGOs and pharmaceutical companies can connect and share knowledge NORWALK, CT (July 31, 2018) — The Duchenne muscular dystrophy (DMD) community now has a dedicated online platform where they can connect […]
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 […]
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 […]
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.
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