The webinar will review the Italfarmaco Givinostat development program for Duchenne and Becker Muscular Dystrophy. Content that will be covered, includes: Updates from the EPIDYS Study, a phase 3 clinical trial of Givinostat in ambulatory boys with DMD. Data from Italfarmaco’s long term studies of Givinostat in DMD populations. 30 minute Q & A. Speakers: […]
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 as gene therapies are emerging. Therefore, it is critical that families accurately understand the process and how they can help support—and possibly benefit from—the development […]
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 the currently enrolling global Phase 3 PolarisDMD trial An introduction to the open-label extension GalaxyDMD trial Results from the Phase 2 MoveDMD trial Click here […]
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 recorded on December 11. This free web broadcast featured a dialog between three physician experts and an experienced Duchenne parent/caregiver. Participants were able to ask […]
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 pre-clinical development of potential therapies to treat patients with Duchenne muscular dystrophy (DMD). The Webinar will feature Debra Miller, founder and CEO, CureDuchenne and Arthur […]
Recording available – DuchenneXchange.org Hosts Free Webinar to Showcase Features of Online Platform Designed to Unite, Support and Educate Duchenne Community
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 DuchenneXchange. If you were unable to attend, please view the presentation using the following link. Click here to watch the recording of the webinar. You’ll be prompted to […]
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.