Lysosomal Storage Disorders: Advocacy Group Receives FDA Orphan Designations

This is the second Blog Post in a series over the next week that will examine Lysosomal Storage Disorders (LSDs) in the rare disease and orphan drug space. This Blog Post presents an advocacy group that receives two FDA Orphan Drug Designations (ODDs) in 2013 for treatment of rare diseases. The chart below identifies the gene therapy that receives FDA ODD where the sponsor is a rare disease advocacy group.