Research interests include Metabolism and Genetic Disorders, Trace Elements in Health, Mitochondrial Function and Pathology, and Lysosomal Storage Disorders Research.
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study
Genetics in Medicine2018cited by 31position: firstdoi