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Clinical Genetics|June 14, 2019
Genotype-phenotype relationships in mucopolysaccharidosis type I (MPS I): Insights from the International MPS I RegistryLorne A Clarke, Roberto Giugliani, Nathalie Guffon, et al.
Molecular Genetics and Metabolism|March 6, 2007
The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type IGregory M Pastores, Pamela Arn, Michael Beck, et al.
Pediatrics|January 2, 2009
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis ILorne A Clarke, J Edmond Wraith, Michael Beck, et al.
Molecular Genetics and Metabolism|October 11, 2025
Unmet needs in the treatment and care of somatic manifestations in mucopolysaccharidosis type II: A targeted literature reviewBarbara K Burton, Daniel Fertek, Peter S Chin, et al.
JIMD Reports|February 12, 2014
Enzyme Replacement Therapy in Mucopolysaccharidosis II Patients Under 1 Year of AgeChristina Lampe, Andrea Atherton, Barbara K Burton, et al.
Molecular Genetics and Metabolism|November 13, 2025
Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome SurveyJoseph Muenzer, Jaco Botha, Hernan Amartino, et al.
Molecular Genetics and Metabolism|August 26, 2022
Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized studyJoseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
Molecular Genetics and Metabolism|August 12, 2022
Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis IIJoseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
Molecular Genetics and Metabolism|April 5, 2026
Cerebrospinal fluid heparan sulfate as a biomarker for neuronopathic mucopolysaccharidoses: Rationale and regulatory challengesJoseph Muenzer, Mark Dant, Patricia I Dickson, et al.
Orphanet Journal of Rare Diseases|May 4, 2017
Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registryJoseph Muenzer, Simon A Jones, Anna Tylki-Szymańska, et al.
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