CureDuchenne was pleased to host the “New Duplication Exon 2 Mouse Model: A Tool for Studies of Duplication Skipping for Duchenne” a webinar with Dr. Kevin Flanigan on November 21, 2013. The webinar was an opportunity for parents to learn about the duplication mutation research and an exciting exon skipping method using AAV delivery with U7 from Dr. Kevin Flanigan at Nationwide Children’s Hospital. CureDuchenne has provided the lead funding for Dr. Flanigan’s duplication research.
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 of the Office of Medical Policy of FDA’s Center for Drug Evaluation and Research, presented detailed information on the different options available to speed the […]