Literature DB >> 16482534

Congenital disorder of glycosylation type Ia presenting as early-onset cerebellar ataxia in an adult.

Kerrie L Schoffer1, John D O'Sullivan, Jim McGill.   

Abstract

Congenital disorders of glycosylation (CDG) are a recently described, underrecognized group of syndromes characterized biochemically by abnormal glycosylation of serum and cellular glycoproteins. We report a previously undiagnosed adult male who presented with early-onset cerebellar ataxia in the context of mental impairment, peripheral neuropathy, retinopathy, body dysmorphism, cardiomyopathy, and hypogonadism. Newly available screening and genetic testing confirmed the diagnosis as CDG type Ia. This case emphasizes that CDG should be considered as a differential diagnosis for adults with early-onset cerebellar ataxia, particularly in those persons with the aforementioned features, and that undiagnosed cases of childhood ataxia may require reassessment now that testing is available.

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Year:  2006        PMID: 16482534     DOI: 10.1002/mds.20804

Source DB:  PubMed          Journal:  Mov Disord        ISSN: 0885-3185            Impact factor:   10.338


  4 in total

Review 1.  Congenital disorders of glycosylation.

Authors:  Irene J Chang; Miao He; Christina T Lam
Journal:  Ann Transl Med       Date:  2018-12

Review 2.  Endocrine manifestations related to inherited metabolic diseases in adults.

Authors:  Marie-Christine Vantyghem; Dries Dobbelaere; Karine Mention; Jean-Louis Wemeau; Jean-Marie Saudubray; Claire Douillard
Journal:  Orphanet J Rare Dis       Date:  2012-01-28       Impact factor: 4.123

3.  Insufficient ER-stress response causes selective mouse cerebellar granule cell degeneration resembling that seen in congenital disorders of glycosylation.

Authors:  Liangwu Sun; Yingjun Zhao; Kun Zhou; Hudson H Freeze; Yun-Wu Zhang; Huaxi Xu
Journal:  Mol Brain       Date:  2013-12-04       Impact factor: 4.041

4.  29 French adult patients with PMM2-congenital disorder of glycosylation: outcome of the classical pediatric phenotype and depiction of a late-onset phenotype.

Authors:  Marie-Lorraine Monin; Cyril Mignot; Pascale De Lonlay; Bénédicte Héron; Alice Masurel; Michèle Mathieu-Dramard; Catherine Lenaerts; Christel Thauvin; Marion Gérard; Emmanuel Roze; Aurélia Jacquette; Perrine Charles; Claire de Baracé; Valérie Drouin-Garraud; Philippe Khau Van Kien; Valérie Cormier-Daire; Michèle Mayer; Hélène Ogier; Alexis Brice; Nathalie Seta; Delphine Héron
Journal:  Orphanet J Rare Dis       Date:  2014-12-11       Impact factor: 4.123

  4 in total

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