Article
Long-Term Efficacy of T3 Analogue Triac in Children and Adults With MCT8 Deficiency: A Real-Life Retrospective Cohort Study.
The Journal of clinical endocrinology and metabolism - 17 Feb 2022
van Geest Ferdy S, Groeneweg Stefan, van den Akker Erica L T, Bacos Iuliu, Barca Diana, van den Berg Sjoerd A A, Bertini Enrico, Brunner Doris, Brunetti-Pierri Nicola, Cappa Marco, Cappuccio Gerarda, Chatterjee Krishna, Chesover Alexander D, Christian Peter, Coutant Régis, Craiu Dana, Crock Patricia, Dewey Cheyenne, Dica Alice, Dimitri Paul, Dubey Rachana, Enderli Anina, Fairchild Jan, Gallichan Jonathan, Garibaldi Luigi R, George Belinda, Hackenberg Annette, Heinrich Bianka, Huynh Tony, Kłosowska Anna, Lawson-Yuen Amy, Linder-Lucht Michaela, Lyons Greta, Monti Lora Felipe, Moran Carla, Müller Katalin E, Paone Laura, Paul Praveen G, Polak Michel, Porta Francesco, Reinauer Christina, de Rijke Yolanda B, Seckold Rowen, Menevşe Tuba Seven, Simm Peter, Simon Anna, Spada Marco, Stoupa Athanasia, Szeifert Lilla, Tonduti Davide, van Toor Hans, Turan Serap, Vanderniet Joel, de Waart Monique, van der Wal Ronald, van der Walt Adri, van Wermeskerken Anne-Marie, Wierzba Jolanta, Zibordi Federica, Zung Amnon, Peeters Robin P, Visser W Edward
Abstract excerpt
CONTEXT: Patients with mutations in thyroid hormone transporter MCT8 have developmental delay and chronic thyrotoxicosis associated with being underweight and having cardiovascular dysfunction. OBJECTIVE: Our previous trial showed improvement of key clinical and biochemical features during 1-year treatment with the T3 analogue Triac, but long-term follow-up data are needed. METHODS: In this real-life...
Read the complete abstract on PubMed