This is a new proposal requesting partial support for "The New Directions in Biology and Disease of Skeletal Muscle," to be held June 25-July 28, 2018. The “New Directions” meeting began in 2004, and since 2004 this meeting has been held every other year with the goal of promoting translational science for neuromuscular disease. At the time this meeting initiated there were no drugs approved by the FDA to treat neuromuscular disease like muscular dystrophy and spinal muscular atrophy. Since its inception, the purpose of this meeting has been to bring together stakeholders that include academic laboratories, small and large pharmaceutical interests, and patient/family advocacy groups with the mission to promote the development and testing of therapy for neuromuscular disease with the focus on muscle biology. This meeting was developed in response to the MD Care Act, which recognized a need for a neuromuscular disease focused meeting with specific emphasis on translation of basic discovery into clinical trials for those with neuromuscular disease. In 2004, at the first New Directions meeting, we learned about exon skipping and its potential for treating Duchenne Muscular Dystrophy. At that same meeting were the first presentations on the toxic RNA mechanisms underlying Myotonic Dystrophy pathogenesis. The New Directions meeting differs from other topically related meetings because of inclusion of industry and academic attendees as well as heavy emphasis on trainee participation. With drug approvals for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy in 2016 and 2017, the eighth New Directions meeting will include sessions on lessons learned from these trials and discuss the hurdles faced in implementing these therapies into the clinical setting.
|Effective start/end date||4/1/18 → 3/31/19|
- National Institute of Neurological Disorders and Stroke (1R13NS106917-01 REVISED)
Spinal Muscular Atrophy
Duchenne Muscular Dystrophy