In rare diseases such as Duchenne, where the disease population is small, the drug development process can vary from that of more common diseases, and totally new treatment approaches such […]
Join to learn about edasalonexent, a novel oral NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy, regardless of mutation type. This webinar will include: An update on […]
Healthcare professionals, personal caregivers and family members of those with Duchenne muscular dystrophy gathered to learn about new data and best practices for steroid use in Duchenne at a webinar […]
CureDuchenne Webinar with Avidity Biosciences, a privately held biotech company pioneering a new class of precision medicines based upon antibody-oligonucleotide conjugates, to brief the Duchenne community about Avidity’s research in […]
DuchenneXchange, the dedicated online platform built by the Duchenne community for the Duchenne community, hosted a free hour-long webinar for Duchenne community members to learn about the benefits of the […]
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 […]
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 […]
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.