(CNN) — The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease. The groundbreaking ...

Adult patients with beta thalassemia will now have an FDA-approved treatment available with luspatercept-aamt (Reblozyl). The therapy treats the rare inherited blood disorder, which requires patients ...

Credit: Shutterstock. The Company is currently planning a Phase 2 trial of PTG-300 in beta-thalassemia patients which they expect to begin before the end of the year. The Food and Drug Administration ...

Orphan drug designation incentives have helped boost enthusiasm for researching and developing drugs for beta thalassemia, a new review concludes. The review encompassed all beta-thalassemia drugs ...

Sickle cell beta-thalassemia refers to an inherited condition that impacts hemoglobin. People with the condition have different changes in each copy of their hemoglobin gene. One causes red blood ...

"Beta Thalassemia Market"In 2020, there are more than 900 beta-thalassemia major patients and more than 200 beta-thalassemia intermedia patients living in the UK. Emerging therapies for Beta ...

Thalassemia is a common genetic condition affecting red blood cells. Children with severe forms of thalassemia often develop symptoms early in life, but effective treatment can manage the condition.

The groundbreaking treatment can now also be used to treat transfusion-dependent beta thalassemia in people 12 and older. Like sickle cell, beta thalassemia is an inherited blood disorder. The FDA’s ...