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A cost-based-plus pricing approach for repurposed tiratricol in the treatment of Allan-Herndon-Dudley syndrome

Abstract Background Tiratricol, an old thyroid drug, has been repurposed as an orphan drug for Allan-Herndon-Dudley syndrome, a rare genetic disorder affecting thyroid hormone transport. Despite public contributions to its repurposing, the current marketing authorization holder, Egetis Therapeutics,...

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Principais autores: Evert Manders, Sarai Keestra, Wilbert Bannenberg, Vincent van der Wel, Saco de Visser, Carla Hollak
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2026-03-01
Serier:Orphanet Journal of Rare Diseases
Fag:
Drug repurposing
Tiratricol
Allan-Herndon-Dudley syndrome
MCT8-defficiency
Drug pricing
Online adgang:https://doi.org/10.1186/s13023-026-04269-7
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