Pioneering Gene Therapy by UCSF Benioff Children's Hospital Oakland Researchers Demonstrates Therapy Can Eliminate Life-Long Need for Transfusions in Patients with Incurable Blood Disorder

Interim results of clinical trials by investigators at UCSF Benioff Children’s Hospital Oakland and Ann and Robert H. Lurie Children’s Hospital of Chicago reveal that a majority of the 22 patients in two Phase 1/2 studies followed for two years or longer remained free from transfusions.

The results of the trials “Gene Therapy in Patients with Transfusion-Dependent╬▓-Thalassemia,” are published today in the New England Journal of Medicine (NEJM). The results are from two separate, two-year clinical studies using LentiGlobin(r) gene therapy to stop or reduce chronic blood transfusions in patients with transfusion-dependent ╬▓-thalassemia (TDT).

Source link

Related posts

Heart Patients Bond Over Their Life-Saving Devices


U.S. Measles Outbreak Hits 107 Cases in 21 States, D.C.


Substituting Olive Oil for Animal Fats Does a Body Good, Study Suggests


This website uses cookies to improve your experience. We'll assume you're ok with this, but you can opt-out if you wish. Accept Read More

Privacy & Cookies Policy