Search Results - Joseph Muenzer
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Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS) by Joseph Muenzer, Roberto Giugliani, Maurizio Scarpa, Anna Tylki-Szymańska, Virginie Jego, Michael Beck
Published in Orphanet Journal of Rare Diseases (2017-10-01)Get full text
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P132: Clinical characteristics and management of patients with mucopolysaccharidosis II (MPS II) in the United States from the Hunter Outcome Survey* by Barbara Burton, Can Ficicioglu, Helio Pedro, Ravi Pathak, Ian Robinson, Ekaterina Wright, Joseph Muenzer
Published in Genetics in Medicine Open (2023-01-01)Get full text
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Levels of glycosaminoglycans in the cerebrospinal fluid of healthy young adults, surrogate-normal children, and Hunter syndrome patients with and without cognitive impairment by Christian J. Hendriksz, Joseph Muenzer, Adeline Vanderver, Jonathan M. Davis, Barbara K. Burton, Nancy J. Mendelsohn, Nan Wang, Luying Pan, Arian Pano, Ann J. Barbier
Published in Molecular Genetics and Metabolism Reports (2015-12-01)Get full text
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Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey by Joseph Muenzer, Hernan Amartino, Roberto Giugliani, Paul Harmatz, Shuan-Pei Lin, Bianca Link, David Molter, Uma Ramaswami, Maurizio Scarpa, Jaco Botha, Jennifer Audi, Barbara K. Burton
Published in Orphanet Journal of Rare Diseases (2025-07-01)Get full text
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Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study by Joseph Muenzer, Barbara K. Burton, Hernan M. Amartino, Paul R. Harmatz, Luis González Gutiérrez-Solana, Matilde Ruiz-Garcia, Yuna Wu, David Merberg, David Alexanderian, Simon A. Jones
Published in Orphanet Journal of Rare Diseases (2023-11-01)Get full text
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Sanfilippo syndrome: consensus guidelines for clinical care by Nicole Muschol, Roberto Giugliani, Simon A. Jones, Joseph Muenzer, Nicholas J. C. Smith, Chester B. Whitley, Megan Donnell, Elise Drake, Kristina Elvidge, Lisa Melton, Cara O’Neill, MPS III Guideline Development Group
Published in Orphanet Journal of Rare Diseases (2022-10-01)Get full text
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P026: Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II: A phase 1/2 study by Barbara Burton, Joseph Muenzer, Paul Harmatz, Deepa Rajan, Simon Jones, Johanna van den Hout, John Mitchell, Matthew Troyer, Natalie Engmann, Rupa Caprihan, Akhil Bhalla, Imanol Zubizarreta, Arlette Weichert, Peter Chin, Carole Ho
Published in Genetics in Medicine Open (2025-01-01)Get full text
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Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry by Joseph Muenzer, Simon A. Jones, Anna Tylki-Szymańska, Paul Harmatz, Nancy J. Mendelsohn, Nathalie Guffon, Roberto Giugliani, Barbara K. Burton, Maurizio Scarpa, Michael Beck, Yvonne Jangelind, Elizabeth Hernberg-Stahl, Maria Paabøl Larsen, Tom Pulles, David A. H. Whiteman
Published in Orphanet Journal of Rare Diseases (2017-05-01)Get full text
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