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

Leslie B Gordon; Mark W Kieran; Monica E Kleinman; Tom Misteli

doi:10.15252/emmm.201606280

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

EMBO Mol Med. 2016 Jul 1;8(7):685-7. doi: 10.15252/emmm.201606280. Print 2016 Jul.

Authors

Leslie B Gordon¹, Mark W Kieran², Monica E Kleinman³, Tom Misteli⁴

Affiliations

¹ Department of Anesthesiology, Perioperative and Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA Department of Pediatrics, Hasbro Children's Hospital and Warren Alpert Medical School of Brown University, Providence, RI, USA.
² Dana-Farber Boston Children's Cancer and Blood Disorder Center, Dana-Farber Cancer Institute, Boston, MA, USA.
³ Department of Anesthesiology, Perioperative and Pain Medicine, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA.
⁴ National Cancer Institute, NIH, Bethesda, MD, USA.

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.

MeSH terms

Child
Clinical Decision-Making / methods
Clinical Trials as Topic*
Drug Discovery* / methods
Humans
Lamin Type A / genetics
Progeria / drug therapy*
Progeria / genetics
Rare Diseases / drug therapy
Rare Diseases / genetics

Substances

Lamin Type A