Results 131 to 140 of about 12,717 (199)

Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II. [PDF]

J Inherit Metab Dis
Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Rust S, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D.   +14 more
europepmc   +1 more source

Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment. [PDF]

Orphanet J Rare Dis
Yee KS, Alexanderian D, Martin S, Olayinka-Amao B, Whiteman DAH.   +4 more
europepmc   +1 more source

Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan. [PDF]

Orphanet J Rare Dis
Nakamura K, Sakai N, Hossain MA, Eisengart JB, Yamamoto T, Tanizawa K, So S, Schmidt M, Sato Y.   +8 more
europepmc   +1 more source

Characterization of orthopedic manifestations in patients with mucopolysaccharidosis II using data from 15 years of the Hunter Outcome Survey. [PDF]

JIMD Rep
Link B, Botha J, Giugliani R.
europepmc   +1 more source

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

A longitudinal study of neurocognition and behavior in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation [PDF]

, 2019
Ahmed, Alia, Chen, Agnes, Dickson, Patricia, Eisengart, Julie B, King, Kelly, Ou, Li, Rudser, Kyle, Shapiro, Elsa, Whitley, Chester B   +8 more
core   +1 more source

Mucopolysaccharidosis II (Hunter's syndrome)

International Bulletin of Otorhinolaryngology, 2019
openaire   +2 more sources

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

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

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