Last Updated: 09/26/2026
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Found 5 publications
Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort.
Journal: Journal of neuromuscular diseases
Published: October 10, 2022
Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort.
Journal: Cardiology in the young
Published: August 23, 2021
Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: January 24, 2019
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.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: July 25, 2017
Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry.
Journal: Molecular genetics and metabolism
Published: February 26, 2014
Showing 1-5 of 5
Last Updated: 09/26/2026