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JACC. Case Reports|January 5, 2022
Mucopolysaccharidosis Type I Diagnosed by Aortic and Mitral Valve ReplacementDalton J Sherwood, Michael C Adams, Anthony J Mazzella, et al.
Human Gene Therapy|May 9, 2015
High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% SalineZheng Fan, Keith Kocis, Robert Valley, et al.
Molecular Genetics and Metabolism|July 14, 2022
Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter studyPaul Harmatz, Joseph Muenzer, Fatih Ezgü, et al.
American Journal of Medical Genetics. Part A|July 23, 2022
Growth in individuals with attenuated mucopolysaccharidosis type I during untreated and treated periods: Data from the MPS I registryLynda E Polgreen, Luisa Bay, Lorne A Clarke, et al.
The Journal of Gene Medicine|June 21, 2005
Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblastsAdelaide Friso, Rosella Tomanin, Sabrina Alba, et al.
Molecular Genetics and Metabolism|July 12, 2021
A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type AFrits A Wijburg, Chester B Whitley, Joseph Muenzer, et al.
JIMD Reports|December 4, 2025
Female Patients With Mucopolysaccharidosis II (MPS II): Insights From the Hunter Outcome SurveyBarbara K Burton, Hernan Amartino, Roberto Giugliani, et al.
BMC Health Services Research|June 6, 2026
An international observational study on transition of care from paediatric to adult services for patients with mucopolysaccharidosis IIKarolina M Stepien, Olulade Ayodele, Joseph Muenzer, et al.
Molecular Genetics and Metabolism|July 17, 2024
Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic MucopolysaccharidosesJoseph Muenzer, Carole Ho, Heather Lau, et al.
Molecular Genetics and Metabolism Reports|June 27, 2017
Levels of glycosaminoglycans in the cerebrospinal fluid of healthy young adults, surrogate-normal children, and Hunter syndrome patients with and without cognitive impairmentChristian J Hendriksz, Joseph Muenzer, Adeline Vanderver, et al.
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