Bleeding Disorders
News
Gene therapy seems safe, effective in hemophilia B
The gene therapy SPK-9001 has produced positive results in a phase 1/2 trial of adults with hemophilia B, according to researchers. SPK-9001...
From the Journals
Gene therapy found promising in patients with hemophilia B
The treatment prevented bleeding and virtually eliminated the need for exogenous factors.
Conference Coverage
Empagliflozin’s heart failure benefits linked to volume drop
ANAHEIM, CALIF. – About half of the observed benefit was tied to a rise in average hematocrit level.
Clinical Review
Immune Thrombocytopenia
Immune thrombocytopenia (ITP) is a common acquired autoimmune disease characterized by low platelet counts and an increased risk of bleeding. The...
News
FDA grants priority review to NDA for avatrombopag
The US Food and Drug Administration (FDA) has granted priority review to the new drug application (NDA) for avatrombopag. Avatrombopag is a...
News from the FDA/CDC
FDA grants orphan drug status to rofecoxib for hemophilic arthropathy
An nonopioid treatment for hemophilic arthropathy could be on the way.
News
FDA approves first-in-class drug for hemophilia A with Factor VIII
The new treatment is given as a weekly injection.
News
Product approved to treat patients with hemophilia A and inhibitors
The US Food and Drug Administration (FDA) has approved use of emicizumab-kxwh (Hemlibra®), a bispecific factor IXa- and factor X-directed antibody...
News
CHMP backs therapy for hemophilia A
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for rurioctocog alfa...
News
CHMP recommends approval of romiplostim in kids
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the approved indication for...
News
Drug receives orphan designation for treatment of PNH
The US Food and Drug Administration (FDA) has granted orphan drug designation to ACH-4471 for the treatment of paroxysmal nocturnal hemoglobinuria...