Literature DB >> 26219880

Friedreich Ataxia in Classical Galactosaemia.

Siobhán Neville1, Siobhan O'Sullivan2, Bronagh Sweeney2, Bryan Lynch3, Donncha Hanrahan4, Ina Knerr1, Sally Ann Lynch5, Ellen Crushell6.   

Abstract

Movement disorders such as ataxia are a recognized complication of classical galactosaemia, even in diet-compliant patients. Here, we report the coexistence of classical galactosaemia and Friedreich ataxia (FRDA) in nine children from seven Irish Traveller families. These two autosomal recessive disorders, the loci for which are located on either side of the centromere of chromosome 9, appear to be in linkage disequilibrium in this subgroup. Both conditions are known to occur with increased frequency amongst the Irish Traveller population.Each member of our cohort had been diagnosed with galactosaemia in the neonatal period, and all are homozygous for the common Q188R mutation in the GALT gene. Eight of the nine patients later presented with progressive ataxia, between the ages of 5-13 years. Another child presented in cardiac failure secondary to dilated cardiomyopathy at 7 years of age. He was not ataxic at presentation and, one year from diagnosis, his neurological examination remains normal. The diagnosis of FRDA was confirmed by detecting the common pathogenic GAA expansion in both alleles of the frataxin gene (FXN) in each patient.Neurological symptoms are easily attributed to an underlying diagnosis of galactosaemia. It is important to consider a diagnosis of Friedreich ataxia in a child from the Irish Traveller population with galactosaemia who presents with ataxia or cardiomyopathy.

Entities:  

Year:  2015        PMID: 26219880      PMCID: PMC4864715          DOI: 10.1007/8904_2015_477

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


  11 in total

1.  Long-term prognosis in galactosaemia: results of a survey of 350 cases.

Authors:  D D Waggoner; N R Buist; G N Donnell
Journal:  J Inherit Metab Dis       Date:  1990       Impact factor: 4.982

Review 2.  Clinical neurogenetics: friedreich ataxia.

Authors:  Abigail Collins
Journal:  Neurol Clin       Date:  2013-11       Impact factor: 3.806

3.  Classical Galactosaemia in Ireland: incidence, complications and outcomes of treatment.

Authors:  K P Coss; P P Doran; C Owoeye; M B Codd; N Hamid; P D Mayne; E Crushell; I Knerr; A A Monavari; E P Treacy
Journal:  J Inherit Metab Dis       Date:  2012-07-03       Impact factor: 4.982

Review 4.  An updated review of the long-term neurological effects of galactosemia.

Authors:  Keith R Ridel; Nancy D Leslie; Donald L Gilbert
Journal:  Pediatr Neurol       Date:  2005-09       Impact factor: 3.372

5.  The impact of galactosaemia on quality of life--a pilot study.

Authors:  C Lambert; A Boneh
Journal:  J Inherit Metab Dis       Date:  2004       Impact factor: 4.982

6.  Genetic basis of transferase-deficient galactosaemia in Ireland and the population history of the Irish Travellers.

Authors:  M Murphy; B McHugh; O Tighe; P Mayne; C O'Neill; E Naughten; D T Croke
Journal:  Eur J Hum Genet       Date:  1999-07       Impact factor: 4.246

Review 7.  Clinical features of Friedreich ataxia.

Authors:  Martin B Delatycki; Louise A Corben
Journal:  J Child Neurol       Date:  2012-06-29       Impact factor: 1.987

8.  Movement disorders in adult patients with classical galactosemia.

Authors:  Ignacio Rubio-Agusti; Miryam Carecchio; Kailash P Bhatia; Maja Kojovic; Isabel Parees; Hoskote S Chandrashekar; Emma J Footitt; Derek Burke; Mark J Edwards; Robin H L Lachmann; Elaine Murphy
Journal:  Mov Disord       Date:  2013-02-11       Impact factor: 10.338

Review 9.  Diagnosis and treatment of Friedreich ataxia: a European perspective.

Authors:  Jörg B Schulz; Sylvia Boesch; Katrin Bürk; Alexandra Dürr; Paola Giunti; Caterina Mariotti; Francoise Pousset; Ludger Schöls; Pierre Vankan; Massimo Pandolfo
Journal:  Nat Rev Neurol       Date:  2009-04       Impact factor: 42.937

10.  Remarkable differences: the course of life of young adults with galactosaemia and PKU.

Authors:  A M Bosch; H Maurice-Stam; F A Wijburg; M A Grootenhuis
Journal:  J Inherit Metab Dis       Date:  2009-10-10       Impact factor: 4.982

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  1 in total

Review 1.  Inherited Metabolic Disorders Presenting with Ataxia.

Authors:  Grace Silver; Saadet Mercimek-Andrews
Journal:  Int J Mol Sci       Date:  2020-08-01       Impact factor: 5.923

  1 in total

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