Hemophilia is characterized by a deficiency in coagulation factors VIII or IX. The general
standard of care for severe hemophilia is frequent intravenous recombinant or plasma …

Stroke in children and young adults is a morbid disease responsible for enormous indirect
societal costs and a high burden of years with disability per affected patient. Recent …

Introduction As gene therapies are incorporated into clinical practice, shared decision‐
making (SDM) is recommended for implementation. Aim To inform development of a …

Background Etranacogene dezaparvovec, the first gene therapy approved for haemophilia B
treatment, was shown to be superior to treatment with continuous prophylactic factor IX in …

Objective: To review efficacy and safety data of valoctocogene roxaparvovec (Roctavian)
and etranacogene dezaparavovec (Hemgenix), novel gene therapies for the treatment of the …

We investigated long-term human coagulation factor IX (huFIX) expression of a novel variant
when delivered into mice and rhesus macaques and compared transduction efficiencies …

Abstract The US Food and Drug Administration (FDA)'s authorization of etranacogene
dezaparvovec (Hemgenix) is a significant milestone, constituting not only the first FDA …

Introduction With recent approval of the first two gene therapies for haemophilia A and B,
educational materials about AAV‐based gene therapy are needed by the haemophilia …

Introduction Etranacogene dezaparvovec is the first gene therapy approved for treatment of
adults with severe and moderately severe hemophilia B. Areas covered This review …

Adeno-associated virus (AAV) gene therapy has been the subject of intensive investigation
for monogenic disease gene addition therapy for more than 25 years, yet few therapies have …