نتایج جستجو برای: mucopolysaccharidosis type

تعداد نتایج: 1343233  

Journal: :International Journal of Case Reports and Images 2015

Journal: :Orphanet Journal of Rare Diseases 2017

Journal: :Clinical science 1979
J J Hopwood V Muller A C Pollard

1. a-L-Iduronidase activity was assayed by incubation of iduronosyl anhydro[ l-3H]mannitol 6-sulphate with homogenates of cultured skin fibroblasts, amniotic cells and leucocytes derived from normal individuals, patients affected with GLiduronidase deficiency disorder (mucopolysaccharidosis type I: Hurler, Scheie and Hurler-Scheie compound) and parents of such patients. 2. The assay for cc-L-id...

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 ...

2013
Bandar Tun Razak

We here describe a videolaryngoscope assisted fibreoptic tracheal intubation in a 17-year-old patient with Hunter Syndrome (Mucopolysaccharidosis Type II) and known difficult intubation who required posterior cervical fusion surgery for cervical canal stenosis. The patient had a history of failed nasal and oral fibreoptic intubation. The use of a videolaryngoscope enabled continuous visualizati...

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