PR001 Gets Fast Track Status for Parkinson Disease With GBA1 Mutation

PR001 Gets Fast Track Status for Parkinson Disease With GBA1 Mutation
The Food and Drug Administration (FDA) has granted Fast Track designation to PR001 (Prevail Therapeutics) for the treatment of Parkinson disease patients with a GBA1 mutation (PD-GBA). PR001 is an investigational single-dose gene therapy that utilizes an AAV9 viral vector to deliver the GBA1 gene to a patient’s cells. Mutations in the GBA1 gene can lead to a deficiency of ... read more
Source: Neurology AdvisorPublished on 2019-07-12By Jennifer Mannheim