Duchenne Expert Webinars

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.

CureDuchenne was pleased to host the “Drug Development and Clinical Trial Participation – A Webinar for the Rare Disease Community” on Friday, September 13, 2013. The webinar was an opportunity for parents to learn about the drug development process – from the pre-clinical phase to the clinical phase to the post approval phase and the regulatory process guiding these phases – from industry experts.

CureDuchenne was honored to host a webinar on February 20, 2013, in collaboration with PPMD and MDA, to discuss accelerated approval for drugs for rare diseases. Dr. Robert Temple, director […]