Bleeding Disorders
Conference Coverage
Study identifies predictors of acquired von Willebrand disease
ATLANTA – Successfully treating Waldenström macroglobulinemia often resolves acquired von Willebrand disease.
News
FDA okays dosing software for hemophilia A
The newly cleared software is designed for use in developing personalized prophylaxis regimens for patients with hemophilia A.
News
FDA okays dosing software for hemophilia A
The newly-cleared software is designed for use in developing personalized prophylaxis regimens for patients with hemophilia A.
News
FDA lifts clinical hold on fitusiran trials
The US Food and Drug Administration (FDA) has lifted the hold on clinical trials of fitusiran, an RNAi therapeutic being developed to treat...
Conference Coverage
VIDEO: Caplacizumab sped platelet response, improved clinical outcomes in acquired TTP
ATLANTA – Phase 2 data on aTTP earned caplacizumab an FDA fast track designation in July 2017.
Conference Coverage
Emicizumab reduces bleeds in kids with hemophilia A and inhibitors
ATLANTA—Updated results from the HAVEN 2 trial have shown that emicizumab prophylaxis can reduce bleeds in children with hemophilia A and factor...
Conference Coverage
Intrabone gene therapy shows promise in beta-thalassemia
ATLANTA – Intrabone gene therapy could offer long-term hope for patients with beta-thalassemia who cannot be treated by allogeneic HSCT.
Conference Coverage
Gene therapy normalized or near-normalized factor VIII in hemophilia A
ATLANTA – Gene therapy is “holy grail” for managing hemophilia – a single-gene disorder with a clear relationship between clotting factor level...
Latest News
HAVEN 2: Emicizumab represents new standard of care for hemophilia A with inhibitors
ATLANTA – Emicizumab safely prevents or substantially reduces bleeds in children with hemophilia A with inhibitors.
Conference Coverage
Polymerization inhibitor improves hemoglobin levels in adolescents with SCD
ATLANTA – Voxelotor demonstrated consistent, sustained improvements in hemoglobin levels in adolescents with sickle cell disease in a phase 2b...
Latest News
Sickle cell: Customized stem cells may improve transfusion efficacy, safety
ATLANTA – Matching red cell products to sickle-cell disease patients with rare Rh antibodies can be done with a little fancy stem cell footwork...