Recent advances in pulmonary arterial hypertension [version 1; referees: 2 approved]

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Title: Recent advances in pulmonary arterial hypertension [version 1; referees: 2 approved]
Authors: Wilkins, M
Aman, J
Harbaum, L
Ulrich, A
Wharton, J
Rhodes, C
Item Type: Journal Article
Abstract: Pulmonary arterial hypertension (PAH) is a rare disorder with a high mortality rate. Treatment options have improved in the last 20 years, but patients still die prematurely of right heart failure. Though rare, it is heterogeneous at the genetic and molecular level, and understanding and exploiting this is key to the development of more effective treatments. BMPR2 , encoding bone morphogenetic receptor type 2, is the most commonly affected gene in both familial and non-familial PAH, but rare mutations have been identified in other genes. Transcriptomic, proteomic, and metabolomic studies looking for endophenotypes are under way. There is no shortage of candidate new drug targets for PAH, but the selection and prioritisation of these are challenges for the research community.
Issue Date: 24-Jul-2018
Date of Acceptance: 9-Jul-2018
ISSN: 2046-1402
Publisher: F1000 Research Ltd
Journal / Book Title: F1000Research
Copyright Statement: © 2018 Wilkins MR et al. This is an open access article distributed under the terms of the Creative Commons Attribution Licence, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Sponsor/Funder: British Heart Foundation
Funder's Grant Number: FS/15/59/31839
Keywords: BMPR2
bone morphogenetic receptor type 2
new drug targets
pulmonary hypertension
Publication Status: Published
Appears in Collections:Department of Medicine
Faculty of Medicine

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