MFF

Clinical Characteristics

The clinical features of this disease, per organ system, include:

Central Nervous System

  • Delayed psychomotor development
  • Developmental regression
  • Poor head and trunk control
  • Absent speech
  • Spasticity
  • Hyperreflexia
  • Epileptic encephalopathy
  • Hypsarrhythmia on EEG
  • Cerebellar atrophy
  • Signal abnormalities in the basal ganglia
  • Peripheral neuropathy
  • Severe hypotonia

Head and Neck

  • Acquired microcephaly
  • Optic atrophy
  • External ophthalmoplegia
  • Poor visual fixation

Laboratory features

  • Increased or normal serum lactate
  • Elongated peroxisomes and elongated mitochondria in fibroblasts
  • Defects in peroxisomal and mitochondrial fission

Other(s)

  • Dysphagia
  • Hepatic steatosis (reported in one patient)