Sobetirome (GC-1) is a thyromimetic drug that binds to the thyroid hormone receptor TRβ1 preferentially compared to TRα1. It has been investigated for the treatment of dyslipidemia, obesity,[1] Pitt–Hopkins syndrome,[2] cholestatic liver disease,[3] multiple sclerosis,[4] bleomycin-induced lung fibrosis,[5] and COVID-19 caused ARDS.[6] It was designated as an orphan drug by the FDA for the treatment of X-linked adrenoleukodystrophy.[7]
^Lammel Lindemann, Jan; Webb, Paul (2016). "Sobetirome: the past, present and questions about the future". Expert Opinion on Therapeutic Targets. 20 (2): 145–149. doi:10.1517/14728222.2016.1090429. ISSN 1744-7631. PMID 26565124.
^Bohlen, Joseph F; Cleary, Colin M; Das, Debamitra; Sripathy, Srinidhi Rao; Sadowski, Norah; Shim, Gina; Kenney, Rakaia F; Buchler, Ingrid P; Banerji, Tapasree; Scanlan, Thomas S; Mulkey, Daniel K; Maher, Brady J (1 August 2023). "Promyelinating drugs promote functional recovery in an autism spectrum disorder mouse model of Pitt–Hopkins syndrome". Brain. 146 (8): 3331–3346. doi:10.1093/brain/awad057. PMC 10393406. PMID 37068912.
^Kosar, Karis; Cornuet, Pamela; Singh, Sucha; Liu, Silvia; Nejak-Bowen, Kari (May 2020). "The Thyromimetic Sobetirome (GC-1) Alters Bile Acid Metabolism in a Mouse Model of Hepatic Cholestasis". The American Journal of Pathology. 190 (5): 1006–1017. doi:10.1016/j.ajpath.2020.01.015. ISSN 0002-9440. PMC 7221298. PMID 32205094.
^Shah, Kamal; Chauhan, Durgesh Nandini; Chauhan, Nagendra Singh; Mishra, Pradeep (13 May 2020). Recent Advancement in Prodrugs. CRC Press. ISBN 978-1-000-05799-7.
^Matralis, Alexios; Karampitsakos, Theodoros; Papaioannou, Ourania; Katsaras, Matthaios; Sampsonas, Fotios; Bouros, Demosthenes; Aidinis, Vassilis; Tzouvelekis, Argyrios (5 September 2021). "Biosynthesis and implementation of Thyroid Receptor beta (TRß) agonists (thyromimetics) for the treatment of pulmonary fibrosis". European Respiratory Journal. 58 (suppl 65): OA4334. doi:10.1183/13993003.congress-2021.OA4334. ISSN 0903-1936. S2CID 244680799.
^Hartley, Pamela. "$100,000 Gift Speeds Exploration of Sobetirome as COVID-19 Treatment". medicine.yale.edu. Retrieved 15 September 2023.
^"Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 15 September 2023.
Sobetirome (GC-1) is a thyromimetic drug that binds to the thyroid hormone receptor TRβ1 preferentially compared to TRα1. It has been investigated for...
reducing toxicity. Thyromimetics selective for TRβ—including eprotirome, sobetirome, resmetirom, and the prodrug VK2809—have been investigated for the treatment...
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