COHORT 1-4 RESULTS FROM A PHASE 1/2 CLINICAL TRIAL OF AN AAV8-MEDIATED LIVER-DIRECTED GENE THERAPY IN ADULTS WITH GLYCOGEN STORAGE DISEASE TYPE IA (GSDIA)
David Rodriguez‐Buritica(The University of Texas Health Science Center at Houston), Eric Crombez(University of California, Los Angeles), Terry G. J. Derks(University Medical Center Groningen), David A. Weinstein(University of Connecticut), John J. Mitchell(University of North Carolina at Chapel Hill), Maria-Luz Couce Pico(Complejo Hospitalario Universitario de Santiago), Ayesha Ahmad(University of Michigan), Rebecca Riba‐Wolman(University of Connecticut), Connie Lee(University of Michigan), Vassili Valayannopoulos(Regeneron (United States))
Cited by 1
Related Papers
Updated International Tuberous Sclerosis Complex Diagnostic Criteria and Surveillance and Management Recommendations
|Pediatric Neurology|2021|727
Phenylalanine hydroxylase deficiency: diagnosis and management guideline
|Genetics in Medicine|2014|718
Proposed guidelines for the diagnosis and management of methylmalonic and propionic acidemia
|Orphanet Journal of Rare Diseases|2014|713
Glycogen Storage Disease Type III diagnosis and management guidelines
|Genetics in Medicine|2010|305
Phenylketonuria Scientific Review Conference: State of the science and future research needs
|Molecular Genetics and Metabolism|2014|271