Applied Cachexia Research, Charité Universitaetsmedizin Berlin, Berlin, Germany.
Respir Med. 2011 Oct;105 Suppl 1:S7-11. doi: 10.1016/S0954-6111(11)70004-X.
Pulmonary arterial hypertension is an orphan disease and a model for drug developments over recent years. Expert centers have focused basic science on the pulmonary vasculature and the right ventricle, followed by a direct transfer of innovative concepts to clinical research. Successful examples for translational experimentation are the endothelin receptor antagonists, prostacyclin receptor agonists, and the activators of soluble guanylate cyclase. On the other hand, there have been failures such as vasoactive intestinal peptide, statins, and escitalopram. Several new drugs and gene therapy are under investigation, thus significant advances are anticipated.
肺动脉高压是一种孤儿病，也是近年来药物开发的一个模型。专家中心将基础科学研究集中在肺血管和右心室上，然后将创新概念直接转化为临床研究。转化实验的成功范例是内皮素受体拮抗剂、前列环素受体激动剂和可溶性鸟苷酸环化酶激活剂。另一方面，也有一些失败的例子，如血管活性肠肽、他汀类药物和依他普仑。目前正在研究几种新药和基因治疗方法，因此预计会有重大进展。
