A single infusion of verbrinacogene setparvovec resulted in normal factor IX levels among all 10 men who received the investigational gene therapy for moderate to severe hemophilia B, study results ...

An investigational, once-weekly prophylactic treatment provided "superior bleeding prevention" as well as normal or near-normal factor VIII activity in patients with severe hemophilia A, according to ...

Single-cell RNA sequencing (scRNA-seq) has transformed the field of transcriptomics by making it possible for researchers to address fundamental questions that could not be tackled by bulk-level ...

VON Willebrand's disease is manifested by a deficiency of factor VIII (antihemophilic factor) and a prolonged bleeding time and is inherited as an autosomal dominant trait of variable penetrance. 1 ...