Healio

Gene therapy potentially curative for patients with transfusion-dependent beta-thalassemia

Please provide your email address to receive an email when new articles are posted on . Thompson described beta-thalassemia as a severe chronic inherited blood disorder due to one of many mutations in ...
MedPage Today

β-Thalassemia Patients Go Transfusion-Free With Gene Therapy

ATLANTA -- Patients with transfusion-dependent β-thalassemia (TDT) who received a gene therapy achieved normal or near-normal levels of total hemoglobin and transfusion independence, with stable iron ...
MedPage Today

Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia

The development and approval of gene therapies has been a potential game changer for patients with beta-thalassaemia who have been dependent on regular red blood cell transfusions -- a substantial ...
WRAL

Gene therapy helps patients avoid blood transfusion

Memories of classical ballet performances dance in Wanda Sihanath's head when she thinks about her childhood in Elgin, Illinois. She remembers balancing at the barre to Beethoven and pirouetting to ...
Monthly Prescribing Reference

Gene Therapy Casgevy Approved for Transfusion-Dependent Beta Thalassemia

At the time of the interim analysis, 91.4% (32/35) of patients achieved transfusion independence for 12 consecutive months. The Food and Drug Administration (FDA) has approved Casgevy ™ (exagamglogene ...
Business Wire

Long-Term Data Presented at ASH Support beti-cel as a Potentially Curative Gene Therapy for β-Thalassemia Patients Who Require Regular Transfusions Through Achievement of ...

All four parent studies are complete, and all patients have transitioned to a long-term follow-up study (LTF-303) SOMERVILLE, Mass.--(BUSINESS WIRE)--Updated data from bluebird bio inc.’s (NASDAQ: ...
News Medical

Gene therapy leads to durable transfusion independence in youngest patients with thalassemia

Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an ...
Nature

Blood Transfusion and Sickle Cell Disease Research

Blood transfusion therapy remains an indispensable intervention in the management of sickle cell disease (SCD), mitigating life‐threatening complications such as vaso‐occlusive crises and stroke. The ...
ascopubs.org

Transfusion-related cost and time burden offsets in patients with myelofibrosis treated with pacritinib compared to best available therapy based on PERSIST-2 trial.

Differential and cumulative impact of metabolic syndrome traits on cardiovascular, renal, and mortality outcomes in cancer patients. This is an ASCO Meeting Abstract from the 2025 ASCO Annual Meeting ...
The American Journal of Managed Care

Evidence Unclear About Superiority of Exchange Transfusion Types in Sickle Cell Disease

A systematic review and meta-analysis found no clear advantage for automated red cell exchange. For patients with sickle cell disease (SCD), exchange transfusion has benefits over simple transfusion ...
News Medical

Gene therapy may be a curative treatment option for patients with transfusion-dependent beta-thalassemia

A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial ...