Hemophilia

Gene Therapy for Hemophilia B Shows Promise

August 01, 2022

A small study finds that an adeno-associated virus gene therapy eliminated both spontaneous bleeding and the need for factor IX prophylaxis in patients with hemophilia B.

ICER Draft Report Finds Beti-Cel is Cost-Effective for Blood Disorder

April 13, 2022

ICER said Bluebird Bio could use an outcome-based pricing plan of five yearly payments totaling $2.1 million for patients treated with beti-cel who no longer need blood transfusions.

Study: Thrombotic Events Increase Mortality Risk for People With Polycythemia Vera or Essential Thrombocythemia

March 11, 2022

Study adds to the evidence that reducing the risk of blood clots should be one of the main goals of managing these myeloproliferative disorders. Monitoring cholesterol levels may be especially important.

New Hemophilia Treatments Look to Cut Number of Infusions

May 12, 2021

Longer half-lives mean less frequent administration and improved quality of life.