Featuring: Jennifer Schneiderman, MD
A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia. Study co-author Jennifer Schneiderman, MD, discusses results, published in the New England Journal of Medicine.
“It's been actually really inspiring to see these patients…because they were just so hopeful and so brave for embarking on this journey, seeing how their lives have been changed and not really being chained to their medical home as much as they had been. Seeing them be able to get away from their thalassemia has really been amazing.”
— Jennifer Schneiderman, MD
For patients with beta-thalassemia major, the only curative treatment so far has been allogeneic hematopoietic stem cell transplant, which requires finding a compatible donor and runs the risk of graft versus host disease. Jennifer Schneiderman, MD, co-author of the new study, discusses her involvement in a phase 3 clinical trial, recently published in the New England Journal of Medicine, in which patients underwent a novel gene therapy with remarkable results, eliminating the need for an allogeneic hematopoietic stem cell transplant.
Recorded on April 13, 2022
This podcast was originally recorded in the Northwestern University Feinberg School of Medicine News Center on April, 2022.
Jennifer Schneiderman, MD associate professor of Pediatrics in the Division of Hematology, Oncology and Stem Cell Transplantation
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