Beta-Thalassemia Gene Therapy Continues to Show Efficacy in Longer-Term Follow-up
Updated results from the phase I/II HGB-204 and HGB-207 trials indicated that several patients with severe, transfusion-dependent beta-thalassemia have remained transfusion-free up to four...
Demystifying Genetic Testing for Bleeding Disorders
When hemophilia and von Willebrand disease (vWD) were first recognized, doctors didn’t diagnose a patient until he (or, much more infrequently, she) experienced a...
Xarelto Manufacturers Settle Lawsuits Over Bleeding Risks
Johnson & Johnson and Bayer, the manufacturers of rivaroxaban (sold as Xarelto), have agreed to pay $775 million to settle thousands of lawsuits over...
EU Panel Recommends Approving Gene Therapy for Thalassemia
Europe’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of Bluebird Bio’s lentiglobin gene-therapy product for patients with transfusion-dependent beta-thalassemia. CHMP is...
New Treatment Approved for Hemophilia A
The U.S. Food and Drug Administration approved turoctocog alfa pegol, an extended half-life factor VIII (FVIII) molecule formerly known as N8-GP, for the treatment...
Alemtuzumab Plus Myeloablative Conditioning Reduces Post-Transplant GVHD Risk
For younger patients with sickle cell disease (SCD) who are eligible for a hematopoietic cell transplantation (HCT), adding alemtuzumab lowered the risk of graft-versus-host...
Congress Passes Bipartisan Sickle Cell Bill
The U.S. Congress recently passed the Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2018, bipartisan legislation...
FDA Approves New Hemophilia A Treatment
The U.S. Food and Drug Administration (FDA) approved turoctocog alfa pegol, formerly known as N8-GP, for the treatment of children and adults with hemophilia...
Caplacizumab Improves Platelet Normalization Time, Reduces Mortality in Acquired TTP
Adding caplacizumab to standard of care for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) shortened the time to the normalization of platelet counts...
Roche Will Donate Hemophilia A Drug to Developing Countries
Roche will donate its new hemophilia A drug emicizumab-kxwh to the World Hemophilia Federation (WHF) Humanitarian Aid Program. Roche joins other companies – including...
Current Issue
April 2019, Volume 5, Issue 5
This issue features a look at the pitfalls of presenting and interpreting clinical trial data, the latest advances in transplant presented at the 2019 TCT Meetings, and more.
