The development of oral biologics and early aggressive treatment to change the course of the disease are among the possibilities, according to Andrew Blauvelt, MD, MBA.
A phase 1 study showed robust responses in patients with TTR amyloidosis with cardiomyopathy, similar to those reported last year in amyloid polyneuropathy.
One infusion of an investigational gene editing therapy could eliminate red blood cell transfusions in beta-thalassemia and severe vaso-occlusive crises in sickle cell disease.