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Jamalpoor A, van Gelder CA, Yousef Yengej FA, Zaal EA, Berlingerio SP, Veys KR, Pou Casellas C, Voskuil K, Essa K, Ammerlaan CM, Rega LR, van der Welle RE, Lilien MR, Rookmaaker MB, Clevers H, Klumperman J, Levtchenko E, Berkers CR, Verhaar MC, Altelaar M, Masereeuw R, Janssen MJ (2021) Cysteamine-bicalutamide combination therapy corrects proximal tubule phenotype in cystinosis

( A ) Resmetirom, a synthetic, liver-targeted, selective thyroid-hormone-receptor- (THR-) agonist, consists of a tri-substituted benzyl ether core in which (i) two iodine atoms present in T3 are replaced by polar, non-halogen substituents (a trifluoromethyl group and a cyano group), and (ii) the hydrophobic phenolic ring of T3 is replaced by a 3,5-dichloro-4-(4-cyano)phenyl moiety