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 […]
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.
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 […]
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 I get to spend with my son Hawken. On Thanksgiving, I am thankful for all those who continue to fight alongside us for a cure […]
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 The Column Group (TCG) to advance its lead SingleCut CRISPR gene editing program in Duchenne muscular dystrophy and potentially other neuromuscular diseases.  CureDuchenne Ventures helped […]
No matter how long we live, the ones who will have known us the longest are our siblings.  For siblings Wil and Emily Cook, the time they have together is more precious knowing Duchenne muscular dystrophy is a critical factor in Wil’s life, and they make the most of it. Wil and Emily grew up […]
          PTC Therapeutics held a community conference today regarding Ataluren.  Stuart Peltz, President and CEO, PTC Therapeutics, Inc., welcomed and thanked the community as well as provided an overview of the dispute process. The call included a Q&A session with questions that were submitted to the company in advance as well […]
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