A Phase I Study of the Safety of AAV2/8 LSPhGAA in Late-onset Pompe Disease

Administered By

Pediatrics, Medical Genetics

Awarded By

National Institutes of Health

Contributors

Bali, Deeksha Sarihyan Co Investigator
Bond, Jennifer Program Leader
Case, Laura Elizabeth Co Investigator
ElMallah, Mai Co Investigator
Hornik, Christoph Paul Vincent Co Investigator
Kishnani, Priya Sunil Co Investigator
Koeberl, Dwight D. Principal Investigator
Smith, Edward Clinton Co Investigator
Young, Sarah Phyllis Co Investigator

Start/End

September 5, 2017 - June 30, 2021