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Familial writer's cramp: a clinical clue for inherited coenzyme Q10 deficiency.

Matthias AmprosiMichael ZechRuth SteigerWolfgang NachbauerAndreas EigentlerElke R GizewskiMichael GugerElisabetta IndelicatoSylvia Boesch
Published in: Neurogenetics (2020)
The spectrum of coenzyme Q10 (CoQ10) deficiency syndromes comprises a variety of disorders, including a form of autosomal recessive cerebellar ataxia (ARCA2) caused by mutations in the AarF domain-containing kinase 3 gene (ADCK3). Due to the potential response to CoQ10 supplementation, a timely diagnosis is crucial. Herein, we describe two siblings with a novel homozygous ADCK3 variant and an unusual presentation consisting of isolated writer's cramp with adult-onset. Cerebellar ataxia developed later in the disease course and remained stable during the follow-up. This report highlights that ARCA2 should be considered in the differential diagnosis of familial writer's cramp.
Keyphrases
  • early onset
  • intellectual disability
  • replacement therapy
  • copy number
  • genome wide
  • protein kinase
  • muscular dystrophy
  • gene expression
  • climate change
  • transcription factor