Upcoming Webinars


In this webinar, Beth Belluscio, MD, PhD, Pfizer Global Clinical Lead for Rare Neurological Disorders, discusses the Phase 3 study of PF-06939926, a gene therapy for Duchenne muscular dystrophy. Dr. Belluscio answers questions from the Duchenne community about the study including aspects of the design and the criteria for enrollment, followed by a Q&A session […]
In this webinar, Sarepta Therapeutics discusses their patient support program, SareptAssist, intended to support individuals through the process of starting and staying on commercial therapy. Services provided by SareptAssist include personalized case management support, as well as information on insurance benefits, financial assistance options, treatment logistics, infusion options, and ongoing education. Dan Madden, Executive Director […]
Suvodirsen (formerly known as WVE-210201) is an investigational stereopure oligonucleotide being developed as a potential treatment for boys with Duchenne muscular dystrophy with mutations amenable to exon 51 skipping. Wave Life Sciences is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating illnesses. Wave has initiated DYSTANCE 51, a global […]
We will provide an update on the scientific rationale for use of vamorolone, and share some results from our open-label Long Term Extension study. The very important pivotal clinical trial is now seeking participants; we will review information about this trial. ReveraGen was recently awarded a grant to study family and physician preferences regarding the […]
On Tuesday, November 12, 2019, from 4:00 PM to 4:30 PM, we learned about the CureDuchenne Biobank, which launched at our 2019 FUTURES national conference and will continue collecting samples at select CureDuchenne events around the country. Topics and their respective speakers, include: An Introduction to our CureDuchenne Biobank and why we created it | […]
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