• Definition: an X-linked dominant disease caused by a CGG trinucleotide repeat expansion in the FMR1 gene (fragile X mental retardation 1 gene) during oogenesis. This leads to its hypermethylation, which silences the gene and renders it unable to express its physiological gene product.
  • Epidemiology
    • Second most common genetic cause of intellectual disability (after trisomy 21)
    • Most common inherited cause of intellectual disability, as trisomy 21 mostly occurs sporadically
  • Clinical features
    • Intellectual Disability: The most common inherited cause of intellectual disability. It affects males more severely than females.
    • Neuropsychiatric:
      • Autism spectrum disorder features are very common.
      • Anxiety, hyperactivity (ADHD-like symptoms), and impulsive behaviors are characteristic.
    • Physical Examination (Classic Buzzwords):
      • Long, narrow face with a prominent forehead and jaw.
      • Large, everted ears.
      • Macroorchidism (enlarged testes), most apparent after puberty.
    • Connective Tissue Findings:
      • Joint hypermobility.
      • Mitral valve prolapse.
      • Flat feet (pes planus).

Mnemonic

Fragile X: “X-tra large” ears, testes, and face in these patients.