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.
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.
TRiNDS is a specialized contract research organization (CRO) for neuromuscular diseases. It is a full-service CRO with four main pillars of solutions in clinical operations, data management, biostatistics, and study measurements. It is unique with its focus on muscular dystrophies and study outcome developments. It is built on the expertise established with the academic clinical trial network: CINRG. TRiNDS bridges gaps for neuromuscular research by drawing in the key leaders in the field.
This informational webinar highlighted the regulatory process to date, updated the community on Marathon Pharmaceutical’s Expanded Access Program (EAP) for deflazacort and discussed what a potential FDA approval of deflazacort would mean in terms of access to the drug in the United States. Some of those who import versions of deflazacort from abroad may have received an update from their distributor with initial details, which was discussed along with steps deflazacort patients might choose to take to help ensure continued access to U.S. supply of deflazacort if FDA approval is granted.