Bleeding Disorders
News
FDA approves second drug for thrombocytopenia in liver disease
The approval is only for patients with chronic liver disease who are scheduled to undergo either a medical or dental procedure.
News
FA pathway key to DNA repair after CRISPR cutting
New research suggests the Fanconi anemia (FA) pathway plays a key role in repairing double-strand breaks (DSBs) created by CRISPR-Cas9 genome...
From the Journals
Researchers identify potential sickle cell disease target
The identification of heme-regulated inhibitor (HRI) was made using a tailored CRISPR screen of adult human erythroid cells.
Conference Coverage
For men with SCD and priapism, hypoxia may prompt RBC adhesion
WASHINGTON – A newly developed biochip allows in vitro examination of microfluidics in sickle cell disease.
News
FDA issues guidance for hemophilia gene therapy development
The agency issued three disease-specific gene therapy guidances, including for retinal disorders and rare diseases.
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The US Food and Drug Administration (FDA) has released several draft guidance documents on gene therapy.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...