A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase DeficiencyBarbara K. Burton, Anthony Quinn, Patrick Deegan et al.|New England Journal of Medicine|2015Cited by 244
Phenylketonuria in adulthood: A collaborative studyRichard Koch, C. Azen, Barbara K. Burton et al.|Journal of Inherited Metabolic Disease|2002Cited by 215
Newborn screening for X-linked adrenoleukodystrophy in New York State: Diagnostic protocol, surveillance protocol and treatment guidelinesBeth Vogel, Michele Caggana, Sue Bradley et al.|Molecular Genetics and Metabolism|2015Cited by 124
Lack of genotype–phenotype correlations and outcome in MCAD deficiency diagnosed by newborn screening in New York StateGeorgianne L. Arnold, Michele Caggana, Carlos A. Saavedra‐Matiz et al.|Molecular Genetics and Metabolism|2009Cited by 54