Methods of treating hemoglobinopathies

摘要

Methods of alleviating the symptoms of hemoglobinopathies, including, but not limited to, sickle cell disease, β-thalassemia, and hemoglobin H disease are provided. In some embodiments, the methods comprise administering an inhibitor selected from an ERK inhibitor, a MEK inhibitor, and a Raf inhibitor. Methods of inhibiting adhesion of sickle red blood cells to endothelial cells are also provided.

主权项

1. A method of alleviating vaso-occlusion in a patient with a hemoglobinopathy comprising administering to the patient a MEK inhibitor; wherein the patient has experienced at least one vaso-occlusive event, and wherein the MEK inhibitor is selected from U0126, PD98059, PD-334581, GDC-0973, CIP-137401, ARRY-162, ARRY-300, PD318088, PD0325901, CI-1040, BMS 777607, AZD8330, AZD6244, AS703026, RDEA119, and GSK1120212.