Literature DB >> 31035122

Biotinidase deficiency: A treatable cause of opticospinal syndrome in young adults.

V Van Iseghem1, M Sprengers2, J De Zaeytijd3, C J M Sindic4, B Willekens5, B Dermaut6, D Hemelsoet2, G Laureys2.   

Abstract

Diagnosis of biotinidase deficiency is rare and usually made in infancy, through newborn screening or after presenting symptoms. We present the case of 19-year old male with progressive optic atrophy and in a second phase spinal cord syndrome unresponsive to immunosuppressive therapies. After diagnosis of profound biotinidase deficiency, oral biotin substitution was started with partial visual improvement and normalization of gait. This case highlights the possibility of late-onset biotinidase deficiency and its treatable character.
Copyright © 2019 Elsevier B.V. All rights reserved.

Entities:  

Keywords:  Demyelinating disease; Metabolic disease (inherited); Optic nerve; Spinal cord; Visual loss

Year:  2019        PMID: 31035122     DOI: 10.1016/j.msard.2019.04.025

Source DB:  PubMed          Journal:  Mult Scler Relat Disord        ISSN: 2211-0348            Impact factor:   4.339


  3 in total

1.  Shortcutting the diagnostic odyssey: the multidisciplinary Program for Undiagnosed Rare Diseases in adults (UD-PrOZA).

Authors:  Nika Schuermans; Dimitri Hemelsoet; Bart Dermaut; Bruce Poppe; Wim Terryn; Sanne Steyaert; Rudy Van Coster; Paul J Coucke; Wouter Steyaert; Bert Callewaert; Elke Bogaert; Patrick Verloo; Arnaud V Vanlander; Elke Debackere; Jody Ghijsels; Pontus LeBlanc; Hannah Verdin; Leslie Naesens; Filomeen Haerynck; Steven Callens
Journal:  Orphanet J Rare Dis       Date:  2022-05-23       Impact factor: 4.303

2.  Biotinidase deficiency is a rare, potentially treatable cause of peripheral neuropathy with or without optic neuropathy in adults.

Authors:  Elizabeth Kellom; Kimberly Stepien; Gregory Rice; Barry Wolf
Journal:  Mol Genet Metab Rep       Date:  2020-12-15

3.  Severe Distal Motor Involvement in a Non-compliant Adult With Biotinidase Deficiency: The Necessity of Life-Long Biotin Therapy.

Authors:  Géraldine Van Winckel; Diana Ballhausen; Barry Wolf; Melinda Procter; Rong Mao; Patricie Burda; Davide Strambo; Thierry Kuntzer; Christel Tran
Journal:  Front Neurol       Date:  2020-10-26       Impact factor: 4.003

  3 in total

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