نتایج جستجو برای: mucopolysaccharidosis 1

تعداد نتایج: 2754438  

Journal: :Jornal de pediatria 2006
Louise L C Pinto Ida V D Schwartz Ana C S Puga Taiane A Vieira Maria Verônica R Munoz Roberto Giugliani

OBJECTIVE To assess the progression of mucopolysaccharidosis II in 11 Brazilian patients over a 12-month period. METHODS Eleven Brazilian patients with mucopolysaccharidosis II were prospectively studied at the Division of Medical Genetics of Hospital de Clínicas de Porto Alegre. The initial assessment and the assessment at 12 months included: anamnesis, physical examination, abdominal nuclea...

Journal: :International Journal of Clinical Biochemistry and Research 2023

Mucopolysaccharidosis is a lysosomal storage disorder, caused due to deficiency of enzymes required for the breakdown Mucopolysaccharides. These undegraded Mucopolysaccharides accumulate in various tissues and cause characteristic features like neurological deficit, impaired motor function, developmental delay, hearing loss, behavioral problems, corneal clouding, glaucoma, respiratory distress,...

Journal: :Brazilian journal of otorhinolaryngology 2016
Ana Paula Fiuza Funicello Dualibi Ana Maria Martins Gustavo Antônio Moreira Marisa Frasson de Azevedo Reginaldo Raimundo Fujita Shirley Shizue Nagata Pignatari

INTRODUCTION Mucopolysaccharidosis (MPS) is a lysosomal storage disease caused by deficiency of α-l-iduronidase. The otolaryngological findings include hearing loss, otorrhea, recurrent otitis, hypertrophy of tonsils and adenoid, recurrent rhinosinusitis, speech disorders, snoring, oral breathing and nasal obstruction. OBJECTIVE To evaluate the impact of enzymatic replacement therapy with lar...

2012
Prathima Gajula Karthikeyan Ramalingam Dinesh Bhadrashetty

Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a member of a group of inherited metabolic disorders together termed mucopolysaccharidosis (MPSs). It is a rare, X-linked disorder caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase. The prevalence of this syndrome is 1:100,000 births. Insufficient enzyme activity results in accumulation of glycosaminoglycans (GAGS...

2003
Michael Beck

10.1586/14750708.3.1.9 © 200 Since enzyme-replacement therapy has been successfully introduced for patients with Gaucher disease, Fabry disease and mucopolysaccharidosis Type I, the principle of this treatment has also been taken into consideration for individuals who are affected by mucopolysaccharidosis Type VI (Maroteaux–Lamy disease), a rare lysosomal storage disorder with multiple organ an...

Journal: :Brain : a journal of neurology 2018
Rebecca J Holley Stuart M Ellison Daniel Fil Claire O'Leary John McDermott Nishanthi Senthivel Alexander W W Langford-Smith Fiona L Wilkinson Zelpha D'Souza Helen Parker Aiyin Liao Samuel Rowlston Hélène F E Gleitz Shih-Hsin Kan Patricia I Dickson Brian W Bigger

Mucopolysaccharidosis IIIB is a paediatric lysosomal storage disease caused by deficiency of the enzyme α-N-acetylglucosaminidase (NAGLU), involved in the degradation of the glycosaminoglycan heparan sulphate. Absence of NAGLU leads to accumulation of partially degraded heparan sulphate within lysosomes and the extracellular matrix, giving rise to severe CNS degeneration with progressive cognit...

2016
Sean R Cantwell Niles J Batdorf Julie E Adams Steven L Moran

Carpal tunnel syndrome (CTS) is the most common neuropathy of the upper extremity [1, 2]. When disease is bilateral and family history is positive, one should rule out hereditary neuropathies, inborn errors of metabolism such as mucopolysaccharidosis, and underlying systemic disorders such as endocrinopathies, renal disease, and amyloidosis (Table 1) [1-5]. The absence of more Sean R Cantwell1,...

Journal: :Journal of medical genetics 1981
H E Hoyme K L Jones M C Higginbottom J S O'Brien

A child is presented with mucopolysaccharidosis VII (beta-glucuronidase deficiency), bringing to six the number of reported patients with the infantile onset form of this disorder. This patient exhibited the following features, previously unrecognised as part of this syndrome: presentation in the neonatal period, progressive joint contractures, and hydrocephalus. This child's course and data fr...

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