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

Clinical Trial Grant

Duke Scholars

Co Investigator Laura Elizabeth Case Orthopaedic Surgery, Physical Therapy

Co Investigator Priya Sunil Kishnani Pediatrics, Medical Genetics

Co Investigator Mai ElMallah Pediatrics, Pulmonary and Sleep Medicine

Principal Investigator Dwight D. Koeberl Pediatrics, Medical Genetics

Co Investigator Sarah Phyllis Young Pediatrics, Medical Genetics

Co Investigator Deeksha Sarihyan Bali Pediatrics, Medical Genetics

Co Investigator Christoph Paul Vincent Hornik Pediatrics, Critical Care Medicine

Administered By

Pediatrics, Medical Genetics

Awarded By

National Institutes of Health

Start Date

September 5, 2017

End Date

June 30, 2020

Administered By

Pediatrics, Medical Genetics

Awarded By

National Institutes of Health

Start Date

September 5, 2017

End Date

June 30, 2020