Open AccessThe decision‐making process and criteria in selecting candidate drugs for progeria clinical trials
Author(s) -
Gordon Leslie B,
Kieran Mark W,
Kleinman Monica E,
Misteli Tom
Publication year - 2016
Publication title -
embo molecular medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.923
H-Index - 107
eISSN - 1757-4684
pISSN - 1757-4676
DOI - 10.15252/emmm.201606280
Subject(s) - medicine , perioperative , anesthesiology , medical school , gerontology , family medicine , surgery , psychiatry , medical education
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 , [De Sandre‐Giovannoli A, 2003]; Eriksson et al , [Eriksson M, 2003]) and strong patient advocacy (Gordon & Gordon, [Gordon AS, 2014]), progeria has rapidly become a vibrant field of study, attracting a wide range of researchers from basic cell biologists to clinicians.