FDA approves Alhemo for hemophilia A or B with inhibitors
The U.S. Food and Drug Administration (FDA) has approved Novo Nordisk’s concizumab, which will be sold under the brand…
Articles by Lindsey Shapiro, PhD
Study suggests Altuviiio may provide benefit over Hemlibra in preventing bleeds
Preventive treatment with Altuviiio (efanesoctocog alfa) provided better bleed prevention than Hemlibra (emicizumab) in adults and adolescents with…
In trial, giroctocogene fitelparvovec safely prevents hemophilia A bleeds
Nearly two-thirds of hemophilia A patients given a single infusion of the investigational gene therapy giroctocogene fitelparvovec were free…
Elocta found superior to standard half-life products: Real-world study
Real-world use of the extended half-life factor replacement therapy Elocta (efmoroctocog alfa) was associated with better bleed control than…
Enhanced FVIII hemophilia A gene therapy may control bleeds better
A gene therapy designed to provide hemophilia A patients with a highly functional version of factor VIII (FVIII), the…
Team develops gene-editing strategy that may be safer, more efficient
A team of U.S. researchers has developed a new gene-editing strategy that could make the approach safer and more efficient…
Acquired hemophilia A high in Hong Kong; most patients older, frail
The incidence of acquired hemophilia A (AHA) was nearly twice as high in Hong Kong than in Caucasian populations…
Biomarin limits Roctavian development to US, Germany, Italy
In an effort to cut costs and boost profitability, Biomarin Pharmaceutical is limiting commercial development of hemophilia A…
NICE reverses reimbursement recommendation for Hemgenix
In a reversal of a previous recommendation, the U.K.’s National Institute for Health and Care Excellence (NICE) has recommended that…
Hemophilia B gene therapy Beqvez offers new choice: Pfizer official
Pfizer is preparing to launch its hemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt) after securing a U.S. approval…