Dr. Gilmore O’Neill, Chief Medical Officer of Sarepta Therapeutics, Inc, gave a scientific overview of Sarepta’s RNA-targeted exon skipping technologies, PMO and PPMO. Dr. O’Neill provided valuable information on this groundbreaking science. If you were unable to participate, please use the link below to view the recording. Click here to watch the recording of the webinar. You’ll be […]
CureDuchenne hosted a webinar with Dr. Paula Clemens and Lauren Morgenroth on the NS Pharma Exon 53 Skipping Program. The webinar was an opportunity for the Duchenne community to learn the most recent status updates, as well as future plans for the science. Click here to watch the recording of the webinar. You’ll be prompted to enter your […]
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.
In a webinar on November 16, 2017, Dr. Eric Olson from University of Texas Southwestern Medical Center explained CRISPR technology and gene therapy for Duchenne.
This webinar provided an update from Santhera Pharmaceuticals about the SIDEROS trial of idebenone in boys taking steroids. Santhera also provided an update about their regulatory efforts to bring this treatment to boys with Duchenne in the US and Europe.