Bleeding Disorders
Feature
Pfizer launches phase 3 gene therapy study in hemophilia B
Fidanacogene elaparvovec is an investigational gene therapy aimed at allowing hemophilia B patients to live free of routine infusions.
News
FDA releases guidance docs on gene therapy
The US Food and Drug Administration (FDA) has released several draft guidance documents on gene therapy. Three are disease-specific guidances—for...
News
CHMP recommends rVWF for VWD
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for vonicog alfa (...
News
CHMP backs approval of caplacizumab
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of caplacizumab (Cablivi) for the...
Conference Coverage
Survey reveals patient perceptions of ITP
STOCKHOLM—A new survey has revealed patients’ perceptions of immune thrombocytopenia (ITP) and how the condition impacts their quality of life (...
Conference Coverage
Drug is convenient alternative for PNH, doc says
STOCKHOLM—Results of a phase 3 study suggest the long-acting C5 complement inhibitor ravulizumab produces similar results as eculizumab in...
From the Journals
Hemophilia adherence tied to perception of disease
Adherence to prophylaxis is generally high, but quality of life and disease coping matter.
News
Emicizumab granted priority review for hemophilia A without inhibitors
The US Food and Drug Administration (FDA) has granted priority review for emicizumab (Hemlibra®) for adults and children with hemophilia A without...
News
Emicizumab gets priority review for hemophilia A without inhibitors
The Food and Drug Administration is expected to make an approval decision on emicizumab in October 2018.
Conference Coverage
Perioperative rVWF alone sufficient for some VWD patients
GLASGOW—Recombinant von Willebrand factor (rVWF) alone can be sufficient as perioperative management for some patients with severe von Willebrand...
Conference Coverage
Emicizumab controls bleeding regardless of inhibitors
GLASGOW—Emicizumab prophylaxis provides “clinically meaningful” control of bleeding whether or not patients have factor VIII inhibitors, according...