December 8, 2018
The 3rd in Mission: Cure’s patient webinar series, this webinar will feature co-directors Megan Golden and Linda Martin. who will discuss Mission: Cure’s strategy for developing disease-altering therapies for chronic and recurrent acute pancreatitis within 10 years – from collaborating with precision-medicine companies to identifying gene-specific treatments, as well as repurposing already-approved drugs. They will also discuss a new model for financing this research.
Read Feba’s recap of the 2018 Global Genes RARE Patient Advocacy Summit, held in Irvine, CA, in October. This was the 7th annual gathering of rare disease patients, advocates, and researchers, and the largest gathering of the rare disease community worldwide.
Industry at Large
December 20, 2018
3:00 – 4:00 p.m.
Rare diseases present unique challenges for researchers and companies working towards treatments and cures. Small patient populations; Hard to diagnose or delayed diagnosis due to lack of education and understanding among medical professionals; Poor understanding of the natural history of the disease and its progression without intervention; No prior pathways to follow; Clinical endpoints that are often unclear; and enrollment and retention challenges. To address these special needs, NORD (National Organization for Rare Disorders) has created a Natural Histories Patient Registry Platform, with extensive input from FDA, NIH, patients, organizations and experts in the field. The IAMRARE Registry Platform is a system that allows organizations to rapidly launch a high-quality, customized registry to collect the data needed to define the natural progression of disease and advance product development.
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