The first reports of FA2H-associated neurodegeneration from two unrelated Iranian families

Neurol Sci. 2023 Dec;44(12):4359-4362. doi: 10.1007/s10072-023-06932-4. Epub 2023 Jul 6.

Abstract

Background: NBIA (neurodegeneration with brain iron accumulation) is a diverse collection of neurodegenerative illnesses defined by iron accumulation in the basal ganglia. The fatty acid hydroxylase-associated neurodegeneration, or FAHN, is one of the uncommon subtypes of NBIAs, associated with inherited autosomal recessive mutations in gene coding the membrane-bound fatty acid 2 hydroxylase (FA2H) enzyme.

Cases: Here, we report two cases with FAHN from two unrelated families from Iran confirmed by whole exome sequencing.

Conclusion: FAHN is an uncommon variant of NBIA that may manifest as spastic paraparesis without signs of iron buildup on brain imaging. As a result, it should be taken into account while making a differential diagnosis of the hereditary spastic paraplegia (HSP) syndrome, especially in individuals who lack iron deposits.

Keywords: FAHN; Fatty acid hydroxylase-associated neurodegeneration; NBIA; Neurodegeneration with brain iron accumulation.

MeSH terms

  • Brain / diagnostic imaging
  • Heredodegenerative Disorders, Nervous System* / genetics
  • Humans
  • Iran
  • Iron
  • Mutation / genetics
  • Pantothenate Kinase-Associated Neurodegeneration* / genetics
  • Spastic Paraplegia, Hereditary* / diagnostic imaging
  • Spastic Paraplegia, Hereditary* / genetics

Substances

  • Iron
  • fatty acid alpha-hydroxylase

Supplementary concepts

  • Fatty Acid Hydroxylase-Associated Neurodegeneration