Literature DB >> 6142938

Early manifestations of multiple sulfatase deficiency.

R D Burk, D Valle, G H Thomas, C Miller, A Moser, H Moser, K N Rosenbaum.   

Abstract

We describe two boys, presenting by 1 year of age, with developmental delay from birth, mildly coarse facial features, and hepatomegaly. These clinical features were most suggestive of a mucopolysaccharidosis, particularly MPS II. Biochemical studies, including sulfate incorporation in fibroblasts and lysosomal enzyme analyses in fibroblasts, leukocytes, and serum, showed abnormalities in both sulfatide and mucopolysaccharide metabolism and led to the diagnosis of multiple sulfatase deficiency. With time, both patients developed an ichthyotic rash and profound intellectual deterioration. We conclude that findings in the first year of life in some patients with MSD may closely resemble those in patients with a MPS disorder rather than the late infantile form of metachromatic leukodystrophy, as is classically described. Thus, MSD should be considered in the young patient suspected of having a MPS disorder.

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Year:  1984        PMID: 6142938     DOI: 10.1016/s0022-3476(84)80550-8

Source DB:  PubMed          Journal:  J Pediatr        ISSN: 0022-3476            Impact factor:   4.406


  8 in total

Review 1.  Lysosomal storage disorders in the newborn.

Authors:  Orna Staretz-Chacham; Tess C Lang; Mary E LaMarca; Donna Krasnewich; Ellen Sidransky
Journal:  Pediatrics       Date:  2009-04       Impact factor: 7.124

Review 2.  Astrocytes and lysosomal storage diseases.

Authors:  K V Rama Rao; T Kielian
Journal:  Neuroscience       Date:  2015-05-30       Impact factor: 3.590

3.  Complex care of individuals with multiple sulfatase deficiency: Clinical cases and consensus statement.

Authors:  Rebecca Ahrens-Nicklas; Lars Schlotawa; Andrea Ballabio; Nicola Brunetti-Pierri; Mauricio De Castro; Thomas Dierks; Florian Eichler; Can Ficicioglu; Alan Finglas; Jutta Gaertner; Brian Kirmse; Joerg Klepper; Marcus Lee; Amber Olsen; Giancarlo Parenti; Arastoo Vossough; Adeline Vanderver; Laura A Adang
Journal:  Mol Genet Metab       Date:  2018-01-31       Impact factor: 4.797

4.  Cervical cord compression and severe hydrocephalus in a child with Saudi variant of multiple sulfatase deficiency. Report of case.

Authors:  K R al-Moutaery; A R Choudhury; M O Hassanen
Journal:  Acta Neurochir (Wien)       Date:  1994       Impact factor: 2.216

Review 5.  Neonatal manifestation of multiple sulfatase deficiency.

Authors:  Andreas Busche; Julia B Hennermann; Friederike Bürger; Hans Proquitté; Thomas Dierks; Annabel von Arnim-Baas; Denise Horn
Journal:  Eur J Pediatr       Date:  2008-12-10       Impact factor: 3.183

Review 6.  Lysosomal Storage Diseases-Regulating Neurodegeneration.

Authors:  Rob U Onyenwoke; Jay E Brenman
Journal:  J Exp Neurosci       Date:  2016-04-05

Review 7.  Molecular Biomarkers in Multiple Sclerosis and Its Related Disorders: A Critical Review.

Authors:  Maryam Gul; Amirhossein Azari Jafari; Muffaqam Shah; Seyyedmohammadsadeq Mirmoeeni; Safee Ullah Haider; Sadia Moinuddin; Ammar Chaudhry
Journal:  Int J Mol Sci       Date:  2020-08-21       Impact factor: 5.923

8.  Natural history of multiple sulfatase deficiency: Retrospective phenotyping and functional variant analysis to characterize an ultra-rare disease.

Authors:  Laura A Adang; Lars Schlotawa; Samuel Groeschel; Christiane Kehrer; Klaus Harzer; Orna Staretz-Chacham; Thiago Oliveira Silva; Ida Vanessa D Schwartz; Jutta Gärtner; Mauricio De Castro; Carrie Costin; Esperanza Font Montgomery; Thomas Dierks; Karthikeyan Radhakrishnan; Rebecca C Ahrens-Nicklas
Journal:  J Inherit Metab Dis       Date:  2020-08-20       Impact factor: 4.982

  8 in total

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