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CureDuchenne hosts conversation with Pfizer to discuss opening of first U.S. sites in phase 3 trial of investigational gene therapy for ambulatory patients with Duchenne muscular dystrophy

Newport Beach, Calif. – May 16, 2022 – CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy (DMD), today released a webinar on the upcoming opening of U.S. trial sites for Pfizer’s CIFFREO trial, a phase 3 study in ambulatory boys with DMD to assess the safety and efficacy of an investigational mini-dystrophin gene therapy. In the webinar, Pfizer’s global clinical lead for rare neurological diseases, Beth Belluscio, MD-PhD, presents critical updates on Pfizer’s CIFFREO trial including trial design, safety considerations, key inclusion/exclusion criteria, and more. The presentation is followed by a thoughtful Q&A session with Debra Miller, founder and CEO of CureDuchenne. Pfizer’s Duchenne gene therapy program was acquired from Bamboo Therapeutics, which received funding from CureDuchenne in 2016.

To watch the webinar, visit https://www.cureduchenne.org/all-news/pfizer-ciffreo-webinar

“We’re very excited that Pfizer’s global gene therapy ambulatory study for Duchenne will be progressing and will now include U.S. sites. We look forward to the future with hope for gene therapy as we strive to improve and extend the lives of those living with Duchenne, a devastating disease,” said CureDuchenne Founder and CEO Debra Miller. 

DMD is the most common form of muscular dystrophy, occurring in approximately 1 in 5,000 male births. Those affected with Duchenne lose their ability to walk, feed themselves, and breathe independently and ultimately succumb to heart failure. For more information, visit: www.ciffreoduchennetrial.com and clinicaltrials.gov/ct2/show/NCT04281485.

About CureDuchenne

CureDuchenne is recognized as a global leader in research, patient care and innovation for improving and extending the lives of those with Duchenne muscular dystrophy. CureDuchenne is dedicated to finding and funding a cure for Duchenne by breaking the traditional charitable mold through an innovative venture philanthropy model that funds groundbreaking research, early diagnosis and community education. For more information on how to help raise awareness and funds needed for research, please visit www.cureduchenne.org.


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