The severity of these rarer types of sickle cell disease varies. — Hemoglobin S–beta-thalassemia: When individuals are diagnosed with this form of sickle cell disease, they inherit one sickle cell ...

Picking a growth stock that becomes a big winner over a decade or more can transform any investor’s returns. I know this from ...

The 12-month price targets, analyzed by analysts, offer insights with an average target of $508.38, a high estimate of ...

It’s been almost a year since the Food and Drug Administration approved the first genetic treatments for sickle cell disease.

Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious ...

β-Thalassemia is a genetic disorder characterized by reduced or absent synthesis of the beta chains of hemoglobin, leading to ineffective erythropoiesis and severe anemia. Patients with ...

Vertex’s pipeline also includes non-CF treatment candidates such as exa-cel for beta-thalassemia, VX-147 for APOL1-mediated kidney disease and nonopioid drugs for pain treatment. Vertex reported ...