The Durability of Roctavian and the Need for Corticosteroids

CTTP is rare hereditary blood clotting disorder stemming from a disease-causing mutation in the ADAMTS13 gene. This gene plays a crucial role in producing the ADAMTS13 enzyme, responsible for regulating blood clotting.

Briana Contreras, an editor with Managed Healthcare Executive, spoke with Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, in this month's episode of Tuning in to the C-Suite podcast. The conversation was about how the disparity in diversity and ethnicity in rare disease clinical trials in the U.S. has led to gaps in understanding diseases and conditions, jeopardizing universal health, and increasing the economic burden of healthcare.

Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, spoke to MHE about the current disparities in diversity and ethnicity in rare disease clinical trials in the U.S., as well as what he's seeing in clinical trials for diseases like hemophilia, in specific.

Expensive gene therapy has grabbed the headlines, but three promising drugs that work in the coagulation pathway are in late-stage development.

A hemophilia expert makes some comparisons between the two approved gene therapies for hemophilia.

Roctavian for hemophilia A and Hemgenix for hemophilia B could spare some patients from treatment that involves intravenous infusions of clotting factors several times a week. But a hemophilia expert cautions that the high-priced gene therapies aren’t a cure and that the reprieve won’t last a lifetime.