نتایج جستجو برای: mucopolysaccharidosis 1

تعداد نتایج: 2754438  

Journal: :The Biochemical journal 1985
J J Hopwood H Elliott

Sulphated N-acetylhexosamines have been isolated from human urine and tentatively identified as N-acetylglucosamine 6-sulphate (GlcNAc6S), N-acetylgalactosamine 6-sulphate (GalNAc6S), N-acetylgalactosamine 4-sulphate (GalNAc4S) and N-acetylgalactosamine 4,6-disulphate (GalNAc4,6diS). Urine from mucopolysaccharidosis-Type-IIID, -IVA and -VI patients compared with that from normal individuals con...

Journal: :Clinical chemistry 2005
Ding Wang Bhramara Eadala Martin Sadilek Nestor A Chamoles Frantisek Turecek C Ronald Scott Michael H Gelb

Spots for Screening of Mucopolysaccharidosis I in Newborns, Ding Wang, Bhramara Eadala, Martin Sadilek, Nestor A. Chamoles, Frantisek Turecek, C. Ronald Scott, and Michael H. Gelb (Departments of 1 Chemistry, 3 Pediatrics, and 4 Biochemistry, University of Washington, Seattle, WA; 2 Laboratory of Neurochemistry, Buenos Aires, Argentina; * address correspondence to this author at: Departments of...

2016

The ERNDIM Urine Mucopolysaccharide scheme is aiming at (1) provision of urine samples obtained from confirmed MPS patients to enable laboratories to gain or maintain experience to identify MPS patients and (2) proficiency testing of laboratories providing urine screening of mucopolysaccharidosis. The scheme is organised by Erasmus Medical Centre (Rotterdam, NL) in conjunction with SKML, the Du...

Journal: :Journal of Biological Chemistry 1996

Journal: :International Bulletin of Otorhinolaryngology 2019

Journal: :International Archives of Otorhinolaryngology 2020

Journal: :World journal of pediatrics : WJP 2015
Agnieszka Różdżyńska-Świątkowska Agnieszka Jurecka Joachim Cieślik Anna Tylki-Szymańska

BACKGROUND Mucopolysaccharidosis (MPS) diseases lead to a profound disruption in normal mechanisms of growth and development. This study was undertaken to determine the general growth of children with MPS I and II. METHODS The anthropometric data of patients with MPS I and II (n=76) were retrospectively analyzed. The growth patterns of these patients were analyzed and then plotted onto Polish...

2017
Arunabha Ghosh Elsa Shapiro Stewart Rust Kathleen Delaney Samantha Parker Adam J Shaywitz Adelaida Morte Gillian Bubb Maureen Cleary Tien Bo Christine Lavery Brian W Bigger Simon A Jones

BACKGROUND Mucopolysaccharidosis type III is a progressive, neurodegenerative lysosomal storage disorder for which there is currently no effective therapy. Though numerous potential therapies are in development, there are several challenges to conducting clinical research in this area. We seek to make recommendations on the approach to clinical research in MPS III, including the selection of ou...

Journal: :Brain : a journal of neurology 2015
Carla Martins Helena Hůlková Larbi Dridi Virginie Dormoy-Raclet Lubov Grigoryeva Yoo Choi Alexander Langford-Smith Fiona L Wilkinson Kazuhiro Ohmi Graziella DiCristo Edith Hamel Jerôme Ausseil David Cheillan Alain Moreau Eva Svobodová Zuzana Hájková Markéta Tesařová Hana Hansíková Brian W Bigger Martin Hrebícek Alexey V Pshezhetsky

Severe progressive neurological paediatric disease mucopolysaccharidosis III type C is caused by mutations in the HGSNAT gene leading to deficiency of acetyl-CoA: α-glucosaminide N-acetyltransferase involved in the lysosomal catabolism of heparan sulphate. To understand the pathophysiology of the disease we generated a mouse model of mucopolysaccharidosis III type C by germline inactivation of ...

Journal: :Journal of neuropathology and experimental neurology 2008
Raquel M Walton Sergey G Magnitsky Gabriela S Seiler Harish Poptani John H Wolfe

Cellular transplantation in the form of bone marrow has been one of the primary treatments of many lysosomal storage diseases (LSDs). Although bone marrow transplantation can help central nervous system manifestations in some cases, it has little impact in many LSD patients. Canine models of neurogenetic LSDs provide the opportunity for modeling central nervous system transplantation strategies...

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