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.
CureDuchenne hosted a webinar with Dr. Kevin Flanigan from Nationwide Children’s Hospital on October 12, 2016. The webinar was an opportunity for the Duchenne community to learn the most recent status of Dr. Flanigan’s duplication mutation research. Dr. Flanigan presented new data on his duplication 2 research.
Catabasis Pharmaceuticals, Inc. webinar “MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy”
CureDuchenne and Catabasis Pharmaceuticals, Inc. hosted a webinar “MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy” on Wednesday, June 22, 2016. Dr. Donovan discussed the positive results from Part A of the MoveDMD trial and reviewed the design and inclusion criteria of Part B (Phase 2) that is currently running. The MoveDMD trial is a Phase 1/2 clinical trial of edasalonexent in boys with Duchenne between the ages of 4 and 7. The goal of Part A included evaluation of the safety and tolerability of edasalonexent. Part B will evaluate the safety and efficacy of edasalonexent in boys with Duchenne.
Bamboo Therapeutics is a recently formed biotechnology company focused on advancing therapies for rare pediatric diseases of the central and neuromuscular systems. Bamboo was founded to advance the pioneering laboratory work of Dr. Jude Samulski, PhD, the former Director of the Gene Therapy Center at the University of North Carolina into human clinical trials. “Gene Therapy” describes the idea of introducing new genetic material into cells to correct an existing problem, often due to a disease-causing mutation. During the June 13, 2016 webinar, Dr. Samulski explained the basics of gene therapy and described Bamboo’s approach to developing a treatment for Duchenne; by utilizing a disabled virus to deliver a small, highly optimized version of the dystrophin gene systemically to skeletal and cardiac muscle.