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

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-Giovannoli et al, ; Eriksson et al, ) and strong patient advocacy (Gordon & Gordon, ), progeria has rapidly become a vibrant field of study, attracting a wide range of researchers from basic cell biologists to clinicians.