Results 131 to 140 of about 6,677,368 (199)

Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II. [PDF]

Orphanet J Rare Dis
Yee KS, Lewis S, Evans E, Romano C, Alexanderian D.   +4 more
europepmc   +1 more source

Hematopoietic Stem Cell Transplantation for Patients with Mucopolysaccharidosis II. [PDF]

Biol Blood Marrow Transplant, 2017
Kubaski F, Yabe H, Suzuki Y, Seto T, Hamazaki T, Mason RW, Xie L, Onsten TGH, Leistner-Segal S, Giugliani R, Dũng VC, Ngoc CTB, Yamaguchi S, Montaño AM, Orii KE, Fukao T, Shintaku H, Orii T, Tomatsu S.   +18 more
europepmc   +1 more source

Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage. [PDF]

Hum Gene Ther
Catalano F, Vlaar EC, Dammou Z, Katsavelis D, Huizer TF, Zundo G, Hoogeveen-Westerveld M, Oussoren E, van den Hout HJMP, Schaaf G, Pike-Overzet K, Staal FJT, van der Ploeg AT, Pijnappel WWMP.   +13 more
europepmc   +1 more source

Carpal Tunnel Syndrome in the Setting of Mucopolysaccharidosis II (Hunter Syndrome). [PDF]

Plast Reconstr Surg Glob Open, 2017
Argenta AE, Davit A.
europepmc   +1 more source

Mucopolysaccharidosis Type II—An Unexpected “3 in 1” Family

Pediatrics and Neonatology, 2016
Hsiang-Yu Lin
doaj   +1 more source

Mucopolysaccharidosis II (Hunter's syndrome)

International Bulletin of Otorhinolaryngology, 2019
openaire   +2 more sources

Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. [PDF]

Mol Ther, 2019
Okuyama T, Eto Y, Sakai N, Minami K, Yamamoto T, Sonoda H, Yamaoka M, Tachibana K, Hirato T, Sato Y.   +9 more
europepmc   +1 more source

Early hematopoietic stem cell transplantation in a patient with severe mucopolysaccharidosis II: A 7 years follow-up. [PDF]

Mol Genet Metab Rep, 2017
Barth AL, de Magalhães TSPC, Reis ABR, de Oliveira ML, Scalco FB, Cavalcanti NC, Silva DSE, Torres DA, Costa AAP, Bonfim C, Giugliani R, Llerena JC, Horovitz DDG.   +12 more
europepmc   +1 more source

Early Recognition of Patients with Neuronopathic Forms of Mucopolysaccharidosis Type II in Clinical Practice. [PDF]

Med Sci (Basel)
Buchinskaia NV, Zakharova EY, Vechkasova AO, Chikova IA, Vashakmadze ND, Namazova-Baranova LS, Ivanov DO, Kutsev SI, Kostik MM.   +8 more
europepmc   +1 more source