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Aviles Family
Gabe was a surprise pregnancy! I was 39 and already had three kids when I gave birth to him, and when I was pregnant, all tests came back indicating that […]
Aluffi Family
Joaquin was diagnosed with Duchenne Muscular Dystrophy in early 2019. While this diagnosis was completely devastating it also answered so many questions that we had been struggling with for years. […]
Sarepta Therapeutics’ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sarepta’s Commercial Process Material
Results from the first 11 participants enrolled in Study 9001-103 ENDEAVOR showed robust transduction, delivering mean vector genome copies of 3.87 per nucleus Treated patients achieved mean micro-dystrophin expression levels […]
Don’t Miss CureDuchenne at the Gene Therapy for Muscular Disorders Event
With recent clinical updates from Pfizer, Sarepta and Audenteson their programs in muscular disorders, the timely Gene Therapy for Muscular Disorders arrives as the only meeting dedicated to overcoming the translational […]
Pfizer Letter to the Duchenne Community
Update on Pfizer’s Duchenne gene therapy Phase 3 clinical program In line with our continued efforts to work with the Duchenne community and share information as it becomes available, we […]
Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
Results suggest a highly potent next-generation treatment that could offer greater efficacy with less frequent dosing SRP-5051 dosed monthly at 30 mg/kg delivered mean exon skipping of 10.79% and mean dystrophin […]
CureDuchenne Cares Educational Webinar: A Conversation About Corticosteroids
Join us for a presentation on the clinical development process behind approved therapies and the importance of dose and administration of corticosteroids by Dr. Emma Ciafaloni. This presentation will be […]
CureDuchenne continues commitment to next-generation gene therapy with investment in Code BioTherapeutics
Today, CureDuchenne Ventures is pleased to share that we made a research investment into Code BioTherapeutics and their next-generation gene therapy platform. This is a promising technology aimed at overcoming […]
“DMD Clinical Trials: An Overview” Presentation by TRiNDS
This presentation by Lauren Morgenroth, CGC and Ana Christensen, MPH at TRiNDS is part of the “A Holistic View of Clinical Trials” webinar and covers the following topics: the clinical […]