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Isoprenylcysteine Carboxylmethyltransferase-Based Therapy for Hutchinson–Gilford Progeria Syndrome

27 Jun 2021

Abstract excerpt

High Resolution Image Download MS PowerPoint Slide Hutchinson–Gilford progeria syndrome (HGPS, progeria) is a rare genetic disease characterized by premature aging and death in childhood for which there were no approved drugs for its treatment until last November, when lonafarnib obtained long-sought FDA approval. However, the benefits of lonafarnib in patients are limited, highlighting the need for new...

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PMID: 34471675
DOI: 10.1021/acscentsci.0c01698

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