Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency

This will be an open-label, single-arm, multi-center EAP, designed to provide treatment access in the US to tiratricol for eligible patients with MCT8 deficiency, also known as AHDS. Patients will undergo clinical and safety assessments before tiratricol treatment initiation, during the titration phase and then at approximately 3-6 monthly intervals or more frequently if clinically indicated, following the initiation of tiratricol treatment and until treatment is completed or discontinued. Adverse events (AEs) and serious adverse events (SAEs) will be recorded and reported as per FDA regulations.