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

Authors :
Leslie B Gordon
Mark W Kieran
Monica E Kleinman
Tom Misteli
Source :
EMBO Molecular Medicine, Vol 8, Iss 7, Pp 685-687 (2016)
Publication Year :
2016
Publisher :
Springer Nature, 2016.

Abstract

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, 2003; Eriksson et 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.

Details

Language :
English
ISSN :
17574676 and 17574684
Volume :
8
Issue :
7
Database :
Directory of Open Access Journals
Journal :
EMBO Molecular Medicine
Publication Type :
Academic Journal
Accession number :
edsdoj.08a28a93112a4ea1aed517321f9624f5
Document Type :
article
Full Text :
https://doi.org/10.15252/emmm.201606280