SICKLE CELL ANEMIA

This early-stage trial tested a new drug called PF-07209326 in 52 people, first in healthy volunteers and then in patients with sickle cell disease. The main goal was to check safety and how the body processes the drug. Researchers also looked for any early signs that the drug mi…

This pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…

This early-stage study tested a new drug, Pociredir (FTX-6058), in 45 adults with sickle cell disease to see if it is safe and how the body processes it. The main goal was to check for side effects and measure drug levels in the blood. Researchers also looked at changes in fetal …

This study looked at whether the Montreal Cognitive Assessment (MoCA), a short test, can accurately screen for thinking and memory problems in adults with sickle cell disease. Researchers compared MoCA results to a full set of standard cognitive tests in 65 adults. The goal is to…

This study tested a drug called canakinumab (ACZ885) in 49 children and young adults aged 8-20 with sickle cell anemia. The goal was to see if it could reduce daily pain compared to a placebo. Participants rated their pain daily using a 0-10 scale, and researchers measured change…

This study measured how common pulmonary hypertension (high blood pressure in the lungs) is in adults with sickle cell anemia. Nearly 1,000 participants had heart ultrasounds and blood tests, then were contacted by phone for up to 3 years to track their health. The goal was to un…

This study measured blood vessel function in 49 adults with sickle cell disease to see how it relates to disease severity. Researchers used laser and pulse wave tests to check small and large blood vessels. The goal was to better understand the disease, not to test a new treatmen…