Rescuing BMPR2-driven endothelial dysfunction in PAH: a novel treatment strategy for the future?

Pulmonary arterial hypertension (PAH) may be idiopathic and corresponds to sporadic disease without any familial history or identified risk factors, or heritable when it occurs in the hereditary context; germline mutations in the bone morphogenetic protein receptor type 2 (BMPR2) gene, are detected in 70% of heritable PAH cases (1). Even in sporadic cases, PAH is associated with impaired BMPRII signalling, occurring through small mothers against decapentaplegic (SMAD) proteins (2).