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Genetics in Medicine : Official Journal of the American College of Medical Genetics|May 16, 2019
Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe diseasePriya S Kishnani, James B Gibson, Michael J Gambello, et al.Genetics in Medicine : Official Journal of the American College of Medical Genetics|March 23, 2018
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 studySi Houn Hahn, David Kronn, Nancy D Leslie, et al.Annals of Neurology|March 28, 2012
Evidence-based path to newborn screening for Duchenne muscular dystrophyJerry R Mendell, Chris Shilling, Nancy D Leslie, et al.Cardiology in the Young|August 23, 2021
Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohortBarry J Byrne, Steven D Colan, Priya S Kishnani, et al.Pediatric Research|June 23, 2009
Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe diseasePriya S Kishnani, Deya Corzo, Nancy D Leslie, et al.Genetics in Medicine : Official Journal of the American College of Medical Genetics|July 17, 2020
Participant choices for return of genomic results in the eMERGE NetworkChristin Hoell, Julia Wynn, Luke V Rasmussen, et al.Journal of Personalized Medicine|January 6, 2018
Ethical Considerations Related to Return of Results from Genomic Medicine Projects: The eMERGE Network (Phase III) ExperienceRobyn Fossey, David Kochan, Erin Winkler, et al.Pageof 3