MCT8 deficiency drug withdrawal trial raises questions about lifelong treatment
Disease control
Completed
This phase 3 study looked at what happens when males with MCT8 deficiency (a rare genetic condition affecting thyroid hormone transport) stop taking tiratricol. Twenty participants who had been stable on tiratricol were randomly assigned to either continue the drug or receive a p…
Phase: PHASE3 • Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC