Patients who will be receiving immune effector cell (IEC) therapy for the treatment of their cancer (leukemia) are being asked to participate in this study. IEC can produce a harmful side effect called Cytokine Release Syndrome (CRS). CRS involves a release of a large amount of proteins into the bloodstream. This may cause changes in blood pressure and…
This study is for children and teens who have been diagnosed with Acute Myeloid Leukemia (AML). AML is a cancer of the bone marrow, the spongy tissue inside the large bones of the body where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood cells called blasts. These blast cells crowd out the normal cells of the bone…
HELIOS-A is a global Phase 3 randomized, open-label study designed to evaluate the safety and efficacy of ALN-TTRSC02 in adult patients with hATTR amyloidosis experiencing neurologic symptoms brought on by the disease. The study will also evaluate any changes in quality of life experienced by study participants. ALN-TTRSC02 utilizes the mechanism of RNA…