Literature DB >> 10352942

Is the perinatal lethal form of Gaucher disease more common than classic type 2 Gaucher disease?

D L Stone1, O P van Diggelen, J B de Klerk, J L Gaillard, M F Niermeijer, R Willemsen, N Tayebi, E Sidransky.   

Abstract

In recent years there has been increased recognition of a severe perinatal lethal form of Gaucher disease, the inherited deficiency of lysosomal glucocerebrosidase. We previously reported a case of severe type 2 Gaucher disease which was seen in a medical center in Rotterdam and now present three new cases from two other families seen at the same center. Mutational analyses of these cases revealed two novel mutations, H311R and V398F, located in exons 8 and 9, respectively. The identification of four cases of lethal type 2 Gaucher disease in a single center seems to be a function of increased awareness of this phenotype, rather than of geographic clustering. The actual incidence of lethal type 2 Gaucher disease may be underestimated, as many cases may have been misclassified as collodion babies or hydrops of unknown cause.

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Year:  1999        PMID: 10352942     DOI: 10.1038/sj.ejhg.5200315

Source DB:  PubMed          Journal:  Eur J Hum Genet        ISSN: 1018-4813            Impact factor:   4.246


  9 in total

1.  Perinatal lethal Gaucher disease.

Authors:  Nishad Plakkal; Amuchou Singh Soraisham; Junya Jirapradittha; Alfredo Pinto-Rojas
Journal:  Indian J Pediatr       Date:  2010-10-06       Impact factor: 1.967

2.  Four Gaucher disease type II patients with three novel mutations: a single centre experience from Turkey.

Authors:  Fatma Derya Bulut; Deniz Kör; Berna Şeker-Yılmaz; Özlem Hergüner; Serdar Ceylaner; Ferda Özkınay; Sebile Kılavuz; Neslihan Önenli-Mungan
Journal:  Metab Brain Dis       Date:  2018-04-14       Impact factor: 3.584

Review 3.  The clinical management of Type 2 Gaucher disease.

Authors:  Karin Weiss; Ashley Gonzalez; Grisel Lopez; Leah Pedoeim; Catherine Groden; Ellen Sidransky
Journal:  Mol Genet Metab       Date:  2014-11-14       Impact factor: 4.797

4.  Type 2 Gaucher disease: the collodion baby phenotype revisited.

Authors:  D L Stone; W F Carey; J Christodoulou; D Sillence; P Nelson; M Callahan; N Tayebi; E Sidransky
Journal:  Arch Dis Child Fetal Neonatal Ed       Date:  2000-03       Impact factor: 5.747

Review 5.  Type 2 Gaucher disease: phenotypic variation and genotypic heterogeneity.

Authors:  N Gupta; I M Oppenheim; E F Kauvar; N Tayebi; E Sidransky
Journal:  Blood Cells Mol Dis       Date:  2010-09-28       Impact factor: 3.039

Review 6.  Diagnosing neuronopathic Gaucher disease: New considerations and challenges in assigning Gaucher phenotypes.

Authors:  Emily C Daykin; Emory Ryan; Ellen Sidransky
Journal:  Mol Genet Metab       Date:  2021-01-09       Impact factor: 4.797

7.  Abnormal nonstoring capillary endothelium: a novel feature of Gaucher disease. Ultrastructural study of dermal capillaries.

Authors:  Helena Hůlková; Helena Poupetová; Klaus Harzer; Pramod Mistry; Johannes M F G Aerts; Milan Elleder
Journal:  J Inherit Metab Dis       Date:  2010-01-05       Impact factor: 4.982

8.  The natural history of type 2 Gaucher disease in the 21st century: A retrospective study.

Authors:  Tamanna Roshan Lal; Gurpreet K Seehra; Alta M Steward; Chelsie N Poffenberger; Emory Ryan; Nahid Tayebi; Grisel Lopez; Ellen Sidransky
Journal:  Neurology       Date:  2020-08-06       Impact factor: 9.910

9.  Identification of novel splice site mutation IVS9 + 1(G > A) and novel complex allele G355R/R359X in Type 1 Gaucher patients heterozygous for mutation N370S.

Authors:  Kourtnee Hoitsema; Dominick Amato; Aneal Khan; Sandra Sirrs; Francis Y M Choy
Journal:  Meta Gene       Date:  2016-03-23
  9 in total

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