Passage Bio's gene therapy candidate PBGM01 was granted rare pediatric disease designation by the FDA as a treatment of infantile GM1 gangliosidosis. Passage BIO plans to launch a Phase I/II clinical trial in the fourth quarter for PBGM0.
Rare pediatric disease tag granted for GM1 gangliosidosis gene therapy
Sign up for Specialty Pharmacy SmartBrief
News affecting the specialty pharmacy industry
Get the intelligence you need: news and information that is changing your industry today, hand-curated by our professional editors from thousands of sources and delivered straight to your inbox.