Literature DB >> 23588843

A Clinically Severe Variant of β-Mannosidosis, Presenting with Neonatal Onset Epilepsy with Subsequent Evolution of Hydrocephalus.

A Broomfield1, R Gunny, I Ali, A Vellodi, P Prabhakar.   

Abstract

β-Mannosidosis results from a functional deficiency of the lysosomal enzyme, β-mannosidase. While being a well recognised, naturally occurring disease in both goats and cattle, it is an extremely rare disorder in humans with the first cases only being recorded in 1986. Until now the severity of the human disease has not mirrored that of its bovine or caprine counterparts, whose presentation is typically in the neonatal period with both altered skull morphology and seizures. Human β-mannosidosis has previously appeared to be a more indolent disease, with its only consistent phenotypic feature being developmental delay of varying severity. We report a patient, homozygous for a private mutation, who presented in the immediate perinatal period with seizures and who subsequently developed communicating hydrocephalus at 2 years of age.These are two new phenotypic features for β-mannosidosis. The first being the neonatal onset of the seizures, for while seizures have been seen in 3 out of the previous 20 documented cases, they have never before manifested prior to 6 months of age. However, as in the previous documented cases, the seizures were difficult to control and were associated with severe developmental delay.The second unique feature about this case was the development of communicating hydrocephalus. We discuss the possible mechanisms of its development.In summary, β-mannosidosis must thus now be considered in the differential diagnosis of neonatal onset seizures, and the potential for the development of hydrocephalus should be monitored during subsequent clinical follow-up.

Entities:  

Year:  2013        PMID: 23588843      PMCID: PMC3755546          DOI: 10.1007/8904_2013_227

Source DB:  PubMed          Journal:  JIMD Rep        ISSN: 2192-8304


  19 in total

1.  Variable clinical presentation of lysosomal beta-mannosidosis in patients with null mutations.

Authors:  Rebecca Bedilu; Katherine A Nummy; Alan Cooper; Ron Wevers; Jan Smeitink; Wim J Kleijer; Karen H Friderici
Journal:  Mol Genet Metab       Date:  2002-12       Impact factor: 4.797

2.  Brain MRI findings in patients with mucopolysaccharidosis types I and II and mild clinical presentation.

Authors:  M Gisele Matheus; Mauricio Castillo; J Keith Smith; Diane Armao; Diane Towle; Joseph Muenzer
Journal:  Neuroradiology       Date:  2004-06-17       Impact factor: 2.804

3.  Beta-mannosidosis in two brothers with hearing loss.

Authors:  L Dorland; M Duran; F E Hoefnagels; J N Breg; H Fabery de Jonge; K Cransberg; F J van Sprang; O P van Diggelen
Journal:  J Inherit Metab Dis       Date:  1988       Impact factor: 4.982

4.  Brain and spine MRI features of Hunter disease: frequency, natural evolution and response to therapy.

Authors:  Renzo Manara; Elena Priante; Marco Grimaldi; Lucia Santoro; Luca Astarita; Rita Barone; Daniela Concolino; Maja Di Rocco; Maria Alice Donati; Simona Fecarotta; Anna Ficcadenti; Agata Fiumara; Francesca Furlan; Irene Giovannini; Franco Lilliu; Rodica Mardari; Gabriele Polonara; Elena Procopio; Angelica Rampazzo; Andrea Rossi; Graziolina Sanna; Rossella Parini; Maurizio Scarpa
Journal:  J Inherit Metab Dis       Date:  2011-04-05       Impact factor: 4.982

5.  The frequency of lysosomal storage diseases in The Netherlands.

Authors:  B J Poorthuis; R A Wevers; W J Kleijer; J E Groener; J G de Jong; S van Weely; K E Niezen-Koning; O P van Diggelen
Journal:  Hum Genet       Date:  1999 Jul-Aug       Impact factor: 4.132

6.  Caprine beta-mannosidosis: clinical and pathological features.

Authors:  M Z Jones; J G Cunningham; A W Dade; D M Alessi; U V Mostosky; J R Vorro; J T Benitez; K L Lovell
Journal:  J Neuropathol Exp Neurol       Date:  1983-05       Impact factor: 3.685

7.  beta-mannosidase deficiency in a female infant with epileptic encephalopathy.

Authors:  A Cooper; J E Wraith; W J Savage; M Thornley; M J Noronha
Journal:  J Inherit Metab Dis       Date:  1991       Impact factor: 4.982

8.  Human beta-mannosidase deficiency: biochemical findings in plasma, fibroblasts, white cells and urine.

Authors:  A Cooper; C Hatton; M Thornley; I B Sardharwalla
Journal:  J Inherit Metab Dis       Date:  1988       Impact factor: 4.982

9.  Beta-mannosidosis in twelve Salers calves.

Authors:  B Abbitt; M Z Jones; T R Kasari; R W Storts; J W Templeton; P S Holland; P E Castenson
Journal:  J Am Vet Med Assoc       Date:  1991-01-01       Impact factor: 1.936

10.  Beta-mannosidae deficiency in two mentally retarded girls with intractable seizures.

Authors:  Mathew Punnachalil Cherian
Journal:  Ann Saudi Med       Date:  2004 Sep-Oct       Impact factor: 1.526

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  5 in total

1.  Hereditary β-mannosidosis in a dog: Clinicopathological and molecular genetic characterization.

Authors:  Pompei Bolfa; Ping Wang; Rajeev Nair; Sreekumari Rajeev; Anibal G Armien; Paula S Henthorn; Tim Wood; Mary Anna Thrall; Urs Giger
Journal:  Mol Genet Metab       Date:  2019-08-10       Impact factor: 4.797

2.  Community structure analysis of transcriptional networks reveals distinct molecular pathways for early- and late-onset temporal lobe epilepsy with childhood febrile seizures.

Authors:  Carlos Alberto Moreira-Filho; Silvia Yumi Bando; Fernanda Bernardi Bertonha; Priscila Iamashita; Filipi Nascimento Silva; Luciano da Fontoura Costa; Alexandre Valotta Silva; Luiz Henrique Martins Castro; Hung-Tzu Wen
Journal:  PLoS One       Date:  2015-05-26       Impact factor: 3.240

3.  β-Mannosidosis caused by a novel homozygous intragenic inverted duplication in MANBA.

Authors:  Maria Blomqvist; Marie Falkenberg Smeland; Julia Lindgren; Per Sikora; Hilde Monica Frostad Riise Stensland; Jorge Asin-Cayuela
Journal:  Cold Spring Harb Mol Case Stud       Date:  2019-06-03

4.  Oral manifestation and dental treatment of pediatric patient with beta-mannosidosis: A case report.

Authors:  Mohammad A Alshoraim; Dania E Al Agili
Journal:  SAGE Open Med Case Rep       Date:  2021-12-15

5.  Variant c.2158-2A>G in MANBA is an important and frequent cause of hereditary hearing loss and beta-mannosidosis among the Czech and Slovak Roma population- evidence for a new ethnic-specific variant.

Authors:  Dana Safka Brozkova; Lukas Varga; Anna Uhrova Meszarosova; Zuzana Slobodova; Martina Skopkova; Andrea Soltysova; Andrej Ficek; Jan Jencik; Jana Lastuvkova; Daniela Gasperikova; Pavel Seeman
Journal:  Orphanet J Rare Dis       Date:  2020-08-26       Impact factor: 4.123

  5 in total

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