Literature DB >> 31959364

[Mucopolysaccharidosis: A review].

M Michaud1, N Belmatoug2, F Catros3, S Ancellin3, G Touati4, T Levade5, F Gaches3.   

Abstract

Mucopolysaccharidosis are lysosomal storage diseases, secondary to the accumulation of mucopolysaccharides. Type 1 mucopolysaccharidosis is the most common form and affects between 0.69 and 1.66 newborns per 100,000. The severity of mucopolysaccharidosis is variable with lethal forms in utero and attenuated forms diagnosed in adults. The most common symptoms are short stature, facial dysmorphism, chronic articular pains that can mimic chronic inflammatory rheumatism, axial and peripheral bone involvement, hepatosplenomegaly and an early carpal tunnel. Depending on the type of mucopolysaccharidosis, corneal, cerebral or cardiac involvements are possible. Screening is based on the analysis of urinary glycosaminoglycans. The deficient enzyme assay and the gene analysis confirm the diagnosis. Mucopolysaccharidosis recognition is important for patient management and family screening. In addition, specific enzyme replacement therapy exists for certain types of mucopolysaccharidosis. Role of clinician is important to evoke and diagnose mucopolysaccharidosis.
Copyright © 2019 Société Nationale Française de Médecine Interne (SNFMI). Published by Elsevier Masson SAS. All rights reserved.

Entities:  

Keywords:  Glycosaminoglycanes; Glycosaminoglycans; Hurler; Morquio; Mucopolysaccharidose; Mucopolysaccharidosis; Sanfilippo; Scheie

Mesh:

Year:  2020        PMID: 31959364     DOI: 10.1016/j.revmed.2019.11.010

Source DB:  PubMed          Journal:  Rev Med Interne        ISSN: 0248-8663            Impact factor:   0.728


  1 in total

1.  Limited diagnostic facilities impeding the therapeutic approach of Mucopolysaccharidosis in Bangladesh: a case report.

Authors:  Orindom Shing Pulock; Susmita Dey Pinky; Syeda Humaida Hasan
Journal:  J Int Med Res       Date:  2022-06       Impact factor: 1.573

  1 in total

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