hrp0089fc5.4 | Thyroid | ESPE2018

Thyroid Hormone Analog Therapy in Patients with MCT8 deficiency: the Triac Trial

Groeneweg Stefan , de Coo Rene , van Beynum Ingrid , van der Knoop Marieke , Aarsen Femke , de Rijke Yolanda , Peeters Robin , Visser Theo , Visser Edward

Introduction: Mutations in the thyroid hormone (TH) transporter MCT8 result in MCT8 deficiency, which is characterized by severe intellectual and motor disability and high serum T3 concentrations inducing thyrotoxicity in peripheral tissues. At present, no effective treatment is available, although preclinical studies suggest that the T3 analog Triac is a promising candidate to i) normalize serum T3 levels and thus alleviate the thyrotoxicosis and ii) restore TH signaling in t...