The decision-making process and criteria in selecting candidate drugs for progeria clinical trials

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Abstract

Hutchinson–Gilford progeria syndrome (progeria) is an extremely rare premature aging disease with a population prevalence of 1 in 20 million. Nevertheless, propelled by the discovery of a causal mutation in the lamin A/C gene (LMNA) (De Sandre-Giovannoliet al,2003; Erikssonet al,2003) and strong patient advocacy (Gordon & Gordon,2014), progeria has rapidly become a vibrant field of study, attracting a wide range of researchers from basic cell biologists to clinicians.

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