Recent advances in pulmonary arterial hypertension

TY - JOUR

T1 - Recent advances in pulmonary arterial hypertension

AU - Wilkins, Martin R

AU - Aman, Jurjan

AU - Harbaum, Lars

AU - Ulrich, Anna

AU - Wharton, John

AU - Rhodes, Christopher J

PY - 2018

Y1 - 2018

N2 - 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.

AB - 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.

KW - Journal Article

KW - Review

U2 - 10.12688/f1000research.14984.1

DO - 10.12688/f1000research.14984.1

M3 - SCORING: Review article

C2 - 30079232

VL - 7

ER -