Development of a Splice-Switching Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy
Abstract
Spinal Muscular Atrophy (SMA) is a devastating neurodegenerative disease that is the leading genetic cause of infantile death worldwide. In this proposal, a novel drug will be analyzed and moved closer to the clinic for a treatment for SMA. The objectives include generating data that will be included within a submission to the U.S. Food and Drug Administration, and the culmination of the project is designed to write and submit an Investigational New Drug application.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Oct 29, 2018
- Source ID
- W81XWH1810165
Entities
People
- Christian Lorson
Organizations
- United States Army