Therapeutic Targets for the Congenital Muscular Dystrophies

Children'S Hosp Of Philadelphia, Philadelphia PA

Investigators

Abstract

DESCRIPTION (provided by applicant): This proposal seeks funding for a unique conference devoted to identifying and initiating opportunities for translational research in the congenital muscular dystrophies (CMDs). The CMDs as a group are a significant contributor to childhood onset neuromuscular morbidity, but have received relatively little attention in neuromuscular research. The primary goal of the conference is to address, evaluate and achieve consensus on therapeutic targets in the CMDs by bringing together experts ranging from basic science to clinical trial design in rare disorders, as well as representatives from industry, advocacy and funding agencies (NIH and MDA). A successful outcome will lead to the construction of a roadmap and investment "portfolio" based on the identification of short term, mid term and long range scientific targets and their subsequent development into treatment strategies with strong translational potential towards clinical trials. This conference will be co-sponsored by Cure CMD, a nonprofit advocacy group focused on awareness, education and research support for translational targets in the CMDs.