Experimental drug shows promise for rare genetic syndrome in toddlers
Disease control
Ongoing
This phase 2 trial tests the drug tiratricol (Triac) in 22 young boys (up to 30 months old) with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. The goal is to see if the drug can improve brain development and reduce harmful thyroid effects in the body. Participants…
Phase: PHASE2 • Sponsor: Rare Thyroid Therapeutics International AB • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC