People with hemophilia are hesitant to take the FDA-approved drugs for a variety of reasons, experts say. Pharma and biotech companies have taken notice.
Currently, the only cure for alpha thalassemia is a bone marrow transplant, but the success of gene therapies for related conditions points to a potential new cure.
Using a zebrafish model, researchers developed a method for producing blood stem cells anywhere in the body with the goal of eventually eliminating the need for bone marrow donors.