Long-term efficacy of T3 analogue Triac in children and adults with MCT8 deficiency: a real-life retrospective cohort study.
Ferdy S van GeestStefan GroenewegErica L T van den AkkerIuliu BacosDiana BarcaSjoerd A A van den BergEnrico Silvio BertiniDoris BrunnerNicola Brunetti-PierriMarco CappaGerarda CappuccioKrishna ChatterjeeAlexander D ChesoverPeter ChristianRégis CoutantDana CraiuPatricia CrockCheyenne DeweyAlice DicaPaul DimitriRachana DubeyAnina EnderliJan FairchildJonathan GallichanLuigi R GaribaldiBelinda GeorgeAnnette HackenbergBianka HeinrichTony HuynhAnna KłosowskaAmy Lawson-YuenMichaela Linder-LuchtGreta LyonsFelipe Monti LoraCarla MoranKatalin E MüllerLaura PaonePraveen G PaulMichel PolakFrancesco PortaChristina ReinauerYolanda B de RijkeRowen SeckoldTuba Seven MenevşePeter SimmAnna SimonMarco SpadaAthanasia StoupaLilla SzeifertDavide TondutiHans van ToorSerap TuranJoel VandernietMonique de WaartRonald van der WalAdri van der WaltAnne-Marie van WermeskerkenJolanta WierzbaFederica ZibordiAmnon ZungRobin P PeetersW Edward VisserPublished in: The Journal of clinical endocrinology and metabolism (2021)
Key features were sustainably alleviated in patients with MCT8 deficiency across all ages and highlight the potential of Triac for MCT8 deficiency in real-life.