• Physical abnormalities
  • Abnormal feeding/failure to thrive
  • Developmental delays
  • Organ system failures including hepatic, renal and cardiac failure


  • Cognitive and intellectual deficiency
  • Speech delay
  • Psychomotor delay
  • Coagulation deficiencies; stroke
  • Seizures
  • Peripheral neuropathy
  • Muscle weakness
  • Ophthalmic issues


  • Worsening of peripheral neuropathy
  • Increased muscle weakness; mobility impairment
  • Skeletal malformations apparent (short stature, fixed joints, etc.)
  • Infertility; hormonal insufficiency
  • Cerebellar ataxia

AT-007-1005 Clinical Study

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