Marlene Rabinovitch, MD
Professor of Pediatrics
Stanford School of Medicine
- Rescuing the BMPR2 Pathway: How and Where Can We Intervene?
Knowing that a mutation in the bone morphogenetic protein receptor type 2 (BMPR2) gene is present in most patients with a hereditary form of pulmonary hypertension (PH) has allowed us to propose therapies that might be of value in patients with all forms of PH.1,2 The premise is based on evidence from cultured cells and experimental animals. Taken together, these studies indicate that dysfunction of BMPR2 and related genes leads to an adverse response of the pulmonary circulation to injury, and that this can cause the progressive elevation in pulmonary arterial (PA) pressure and right heart failure in patients with PA hypertension (PAH).