Conditions / Genetic

Lesch-Nyhan syndrome

info · Genetic · ICD-10: E79.1

A purine-pyrimidine metabolic disorder characterized by mental retardation, spastic cerebral palsy, choreoathetosis, uric acid urinary stones, and self-destructive biting of fingers and lips that has_material_basis_in mutation in the HPRT1 gene on chromosome X

A purine-pyrimidine metabolic disorder characterized by mental retardation, spastic cerebral palsy, choreoathetosis, uric acid urinary stones, and self-destructive biting of fingers and lips that has_material_basis_in mutation in the HPRT1 gene on chromosome Xq26.

Signs and symptoms

  • Poor head control
  • Hypotonia
  • Intellectual disability
  • Global developmental delay
  • Hyperuricemia
  • Self-injurious behavior
  • Nephrocalcinosis
  • Short stature
  • Dystonia
  • Motor delay

Medications that may treat it

lesinurad

Also known as: Complete hypoxanthine-guanine phosphoribosyltransferase deficiency; HG-PRT deficiency; HPRT1 deficiency; Hypoxanthine-guanine phosphoribosyltransferase deficiency; Hypoxanthine-guanine-phosphoribosyltransferase deficiency