Once-weekly subcutaneous administration of the novel recombinant, humanized, IgG4 bispecific antibody emicizumab prevents or reduces bleeds in patients with haemophilia A and factor VIII inhibitors in the phase III HAVEN 1* trial, offering a potential new standard of care for this severe bleeding disorder.
Rosuvastatin led to improved coagulation profile, in particular lower levels of factor VIII procoagulant activity (VIII:C), among patients who had venous thrombosis (VT) previously, suggesting that statins could reduce the risk of recurrent VT, according to a study presented at the ISTH 2017 Congress in Berlin, Germany.
Gene-transfer therapy using adeno-associated virus in patients with severe haemophilia A resulted in sustained levels of coagulation factor VIII (FVIII) expression within 1 year of observation, reducing spontaneous bleeds and FVIII infusions in most patients, interim results of a phase 1/2 dose-escalation study have shown.
Idarucizumab, a dabigatran-reversing humanized monoclonal antibody fragment, rapidly, completely, and durably reversed the anticoagulant effect of dabigatran in elderly patients with life-threatening bleeding or undergoing invasive medical interventions, according to a full cohort analysis of the RE-VERSE AD* study presented at the ISTH 2017 Congress in Berlin, Germany.
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Dr Michael Lim, a senior consultant at the Paediatric Pulmonary and Sleep Division, National University Hospital, Singapore, speaks to Roshini Claire Anthony on the rare disease that is cystic fibrosis.
Empagliflozin treatment in patients with type 2 diabetes mellitus exerts beneficial effects on cardiovascular (CV) risk factors, including blood pressure and uric acid, according to a systematic review and meta-analysis.
A 10 or 20 mg/kg daily dose of cannabidiol could almost halve the risk of seizures in children with Dravet syndrome, according to the phase III GWPCARE2* study presented at the American Academy of Neurology annual meeting (AAN 2019).