Duchenne Clinical Trials & Drug Development
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 […]
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.