This study will investigate the effect of treatment with tiratricol (also called Triac) in young boys (≤30 months) with MCT8 deficiency (also called the Allan-Herndon-Dudley syndrome (AHDS)). The hypothesis tested is that treatment with tiratricol will have a beneficial effect on the hypothyroid state in the brain as well as the hyperthyroid state in peripheral organs and tissues in these patients. Patients will initially be treated for 96 weeks with tiratricol, treatment effect on neurodevelopment impairment caused by hypothyroidism and peripheral thyrotoxicosis will be evaluated after 96 weeks treatment. Patients will be offered to continue on treatment for an additional 3 years.
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Gross Motor Function Measure 88 (GMFM 88) total score
Timeframe: 96 weeks, Year 3, Year 4 and Year 5
Bayley Scales of Infant Development III Gross Motor Skill Domain score
Timeframe: 96 weeks, Year 3, Year 4 and Year 5