Internal medicine

Pathology

Fetus

Adverse effect

Clinical endpoint

Placebo

Fetal hemoglobin

Disease

Vaso-occlusive crisis

Biology

Gastroenterology

Surgery

Alternative medicine

Anemia

Acute chest syndrome

Tolerability

Sickle cell anemia

Hemoglobin

Pregnancy

Hemolysis

Clinical trial

Genetics

Medicine

Blood

In a randomized, placebo-controlled, phase 1/2 study in patients with SCD, voxelotor (500-1000 mg per day) was well tolerated. All patients receiving voxelotor for ≥28 days demonstrated hematologic improvements, suggesting disease-modifying activity in SCD.

A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease