Infancy:
Physical abnormalities
Abnormal feeding/failure to thrive
Developmental delays
Organ system failures including hepatic, renal and cardiac failure
Childhood:
Cognitive and intellectual deficiency
Speech delay
Psychomotor delay
Coagulation deficiencies; stroke
Seizures
Peripheral neuropathy
Muscle weakness
Ophthalmic issues
Adulthood:
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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