Results 91 to 100 of about 8,162 (165)

Rescue of neurologic disease in mucopolysaccharidosis type II mice via AAV-mediated liver delivery of brain-penetrating iduronate-2-sulfatase. [PDF]

Neurotherapeutics
Jin X, Ye Q, Wu X, Su J, Song L, Fu J, Xu Q, Luo M, Liu F, Ren C, Hu M, Liu M, An Y, Li Q, Li M, Yang Y.   +15 more
europepmc   +1 more source

iPS-derived neural stem cells for disease modeling and evaluation of therapeutics for mucopolysaccharidosis type II. [PDF]

Exp Cell Res, 2022
Hong J, Cheng YS, Yang S, Swaroop M, Xu M, Beers J, Zou J, Huang W, Marugan JJ, Cai X, Zheng W.   +10 more
europepmc   +1 more source

Newborn Screening Program for Mucopolysaccharidosis Type II and Long-Term Follow-Up of the Screen-Positive Subjects in Taiwan. [PDF]

J Pers Med, 2022
Lin HY, Chang YH, Lee CL, Tu YR, Lo YT, Hung PW, Niu DM, Liu MY, Liu HY, Chen HJ, Kao SM, Wang LY, Ho HJ, Chuang CK, Lin SP.   +14 more
europepmc   +1 more source

Novel Phenotypic Insights into the <i>IDS</i> c.817C>T Variant in Mucopolysaccharidosis Type II from Newborn Screening Cohorts. [PDF]

Int J Neonatal Screen
Beauregard-Lacroix É, Menello C, Steffensen M, Lin HY, Chuang CK, Lin SP, Ficicioglu C.   +6 more
europepmc   +1 more source

A new strategy of desensitization in mucopolysaccharidosis type II disease treated with idursulfase therapy: A case report and review of the literature. [PDF]

Mol Genet Metab Rep, 2022
Gragnaniello V, Carraro S, Rubert L, Gueraldi D, Cazzorla C, Massa P, Zanconato S, Burlina AB.   +7 more
europepmc   +1 more source

Case report of neuronopathic mucopolysaccharidosis type II: Early intracerebroventricular enzyme replacement therapy and hematopoietic cell transplantation with developmental outcomes up to 5 years of age. [PDF]

Mol Genet Metab Rep
Ikari A, Hama A, Okuyama T.
europepmc   +1 more source

Glycosaminoglycan signatures in body fluids of mucopolysaccharidosis type II mouse model under long-term enzyme replacement therapy. [PDF]

J Mol Med (Berl), 2022
Maccari F, Rigon L, Mantovani V, Galeotti F, Salvalaio M, D'Avanzo F, Zanetti A, Capitani F, Gabrielli O, Tomanin R, Volpi N.   +10 more
europepmc   +1 more source

Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis Type II: A Systematic Literature Review. [PDF]

Int J Mol Sci
Al-Hertani W, Pathak RR, Evuarherhe O, Carter G, Schaeffer-Koziol CR, Whiteman DAH, Wright E.   +6 more
europepmc   +1 more source

Real world long-term outcomes in patients with mucopolysaccharidosis type II: A retrospective cohort study. [PDF]

Mol Genet Metab Rep, 2021
Tomita K, Okamoto S, Seto T, Hamazaki T.
europepmc   +1 more source

Oral trehalose improves histological and behavior symptoms of mucopolysaccharidosis type II in iduronate 2-sulfatase deficient mice. [PDF]

Sci Rep
Lee H, Han JH, Jeong RG, Kang YJ, Choi BH, Kim SR, Cheon CK, Hur J, Lee SY.   +8 more
europepmc   +1 more source