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 ...

They’ve been going to UK since 2019. “Noah has a blood disease. It’s called Beta thalassemia major and of all the thalassemia’s that are out there, this one is the worst one, because it ...

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

This therapy treats two blood-related disorders: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Casgevy is approved in several regions: the U.S., the U.K., the ...

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.

The one-time treatment for sickle cell disease and beta thalassemia can generate billions in revenue, but the problem is that it could take a while for the rollout. Analysts at Bloomberg don't ...

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 ...

Exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals) is indicated for 'the treatment of transfusion-dependent β‑thalassemia in patients 12 years of age and older for whom haematopoietic stem ...

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 ...

So far, only 20 patients have been treated with the therapy, which is a functional cure for both sickle cell disease (SCD) and beta thalassemia. Wall Street analysts estimate on average that ...