THALASSEMIA

This early-stage study tested whether a potential new medicine for sickle cell disease, called etavopivat, affects the heart's electrical activity. Researchers gave the drug at different doses to 33 healthy volunteers to check for safety, particularly looking at heart rhythm. The…

This study tested a one-time treatment using the gene-editing tool CRISPR to try to help people with severe thalassemia. Doctors took a patient's own blood stem cells, edited a gene in the lab, and then gave them back. The main goal was to see if this single treatment could allow…

This Phase 2 clinical trial tested an oral medication called etavopivat in 53 patients with sickle cell disease or thalassemia. The study aimed to see if the drug could safely reduce patients' need for blood transfusions and increase their hemoglobin levels. Researchers measured …

This study investigated whether adding the supplement L-glutamine to standard care could lower high blood pressure in the lung arteries of adults with a specific type of thalassemia, a genetic blood disorder. For 60 days, one group took oral L-glutamine while a control group rece…

This pilot study tested a combined transplant from a partially matched family donor. It aimed to treat patients who had both a serious blood disorder (like leukemia) and advanced kidney disease at the same time. The goal was to cure the blood disorder and restore kidney function …

This early study tested a new medicine called etavopivat, which is being developed for sickle cell disease and thalassemia. It involved 24 healthy Chinese volunteers to see how their bodies process a single dose of the drug and to check for any side effects. The main goal was to …

This study aimed to understand how often dangerous iron builds up in the heart of patients who need frequent blood transfusions for conditions like thalassemia, sickle cell disease, or myelodysplasia. Researchers observed 110 patients across 14 centers, using heart and liver MRI …