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The Journal of Pediatrics|May 6, 2004
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase)James E Wraith, Lorne A Clarke, Michael Beck, et al.
Journal of Inherited Metabolic Disease|September 10, 2024
Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis IIJoseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
JIMD Reports|February 25, 2026
Intrathecal idursulfase-IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single-arm, open-label, phase 2/3 substudy and extensionJoseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 27, 2022
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia BPaul Harmatz, Carlos E Prada, Barbara K Burton, et al.
Molecular Genetics and Metabolism|September 12, 2020
Therapy development for the mucopolysaccharidoses: Updated consensus recommendations for neuropsychological endpointsJohanna H van der Lee, Jonathan Morton, Heather R Adams, et al.
Molecular Genetics and Metabolism|May 15, 2017
Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedureJohanna H van der Lee, Jonathan Morton, Heather R Adams, et al.
The New England Journal of Medicine|December 30, 2025
An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis IIJoseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
The Journal of Pediatrics|May 29, 2019
The North Carolina Experience with Mucopolysaccharidosis Type I Newborn ScreeningJennifer L Taylor, Kristin Clinard, Cynthia M Powell, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|December 15, 2010
Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndromeJoseph Muenzer, Michael Beck, Christine M Eng, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|April 22, 2026
A North Carolina Newborn Screening Pilot for Mucopolysaccharidosis II: Evaluating Endogenous Non-Reducing End Glycosaminoglycan Analysis and IDS Sequencing as Higher-Tier Testing OptionsKaterina S Kucera, Kristin Clinard, Samantha L Blake, et al.
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