Hyperekplexia: treatment of a severe phenotype and review of the literature

Can J Neurol Sci. 2011 May;38(3):411-6. doi: 10.1017/s0317167100011793.

Abstract

Hyperekplexia is a rare disorder caused by autosomal dominant or recessive modes of inheritance and characterized by episodes of exaggerated startle. Five causative genes have been identified to date. The syndrome has been recognized for decades and due to its rarity, the literature contains mostly descriptive reports, many early studies lacking molecular genetic diagnoses. A spectrum of clinical severity exists. Severe cases can lead to neonatal cardiac arrest and death during an episode, an outcome prevented by early diagnosis and clinical vigilance. Large treatment studies are not feasible, so therapeutic measures continue to be empiric. A marked response to clonazepam is often reported but refractory cases exist. Herein we report the clinical course and treatment response of a severely affected infant homozygous for an SLC6A5 nonsense mutation and review the literature summarizing the history and genetic understanding of the disease as well as the described comorbidities and treatment options.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Adult
  • Anticonvulsants / therapeutic use
  • Developmental Disabilities / epidemiology
  • Developmental Disabilities / genetics
  • Feeding and Eating Disorders / epidemiology
  • Feeding and Eating Disorders / genetics
  • Female
  • Genetic Testing
  • Humans
  • Infant
  • Muscle Hypertonia* / epidemiology
  • Muscle Hypertonia* / genetics
  • Muscle Hypertonia* / therapy
  • Phenotype*
  • Receptors, Glycine / genetics
  • Reflex, Abnormal / genetics
  • Sleep Apnea Syndromes / epidemiology
  • Sleep Apnea Syndromes / genetics

Substances

  • Anticonvulsants
  • Receptors, Glycine