Results 101 to 110 of about 9,431 (199)

Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates. [PDF]

Mol Ther Methods Clin Dev, 2023
Chen N, Ehmann DE, Crooker R, Derakhchan K, Fang X, Felice B, Galbreath EJ, Glaus C, Gu H, Huang Y, Li C, Li X, Liu N, Palmieri K, Simic D, Sypek J, Thompson S, Winkelmann CT, Choi VW.   +18 more
europepmc   +1 more source

Mucopolysaccharidosis type II (Hunter syndrome) – A case report [PDF]

, 2017
Hunter syndrome or mucopolysaccharidosis (MPS) type II is an X-linked recessive disorder caused by a defect in themetabolism of glycosaminoglycans (GAGs).
Jyotsna, Shrivastava, Amit, Agrawal
core   +1 more source

Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study. [PDF]

Mol Genet Metab, 2022
Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D, HGT-HIT-094 Study Group.   +14 more
europepmc   +1 more source

Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II. [PDF]

Mol Genet Metab, 2022
Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D, SHP609-302 study group.   +14 more
europepmc   +1 more source

Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice. [PDF]

Mol Ther Methods Clin Dev, 2022
Morimoto H, Morioka H, Imakiire A, Yamamoto R, Hirato T, Sonoda H, Minami K.   +6 more
europepmc   +1 more source

Isokinetic strength differences in patients with mucopolysaccharidosis I, II, and VI [PDF]

, 2013
University of Miinesota M.S. thesis. May 2013. Major: Kinesiology. Advisor: Donald R. Dengel, Ph.D. 1 computer file (PDF); v, 42 pages, appendix p.
Taylor, Natalie Ebsch
core  

CORRIGENDUM: Interim results of the first stage of a multicenter open multi-cohort study of the safety, pharmacokinetics, pharmacodynamics and efficacy of veranafusp alfa in adult patients with mucopolysaccharidosis type II. [Pharmacy & Pharmacology. 2026;14(1):81-96. DOI: 10.19163/2307-9266-2026-14-1-81-96]

Фармация и фармакология (Пятигорск)
Related to the article: Lukina E.A., Ponomarev R.V., Trishina S.V., Gabitova E.S., Vashakmadze N.D., Karkashadze G.A., Namazova-Baranova L.S. Interim results of the first stage of a multicenter open multi-cohort study of the safety, pharmacokinetics ...
Editorial Edit
doaj   +1 more source

Clinical Characteristics and Healthcare Resource Utilization for Patients with Mucopolysaccharidosis II (MPS II) in the United States: A Retrospective Chart Review. [PDF]

J Health Econ Outcomes Res, 2022
Ayodele O, Müller K, Setayeshgar S, Alexanderian D, Yee KS.   +4 more
europepmc   +1 more source

Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data. [PDF]

Int J Mol Sci, 2021
Giugliani R, Martins AM, Okuyama T, Eto Y, Sakai N, Nakamura K, Morimoto H, Minami K, Yamamoto T, Yamaoka M, Ikeda T, So S, Tanizawa K, Sonoda H, Schmidt M, Sato Y.   +15 more
europepmc   +1 more source

Impact of the Timing of Enzyme Replacement Therapy Initiation and Cognitive Impairment Status on Outcomes for Patients with Mucopolysaccharidosis II (MPS II) in the United States: A Retrospective Chart Review. [PDF]

J Health Econ Outcomes Res, 2022
Yee KS, Alexanderian D, Feng Y, Ren X, Schweikert B, Ayodele O.   +5 more
europepmc   +1 more source