Gene therapy has proved to be a safe long-term treatment for haemophilia B. The benefits of a single dose persist for at ...

The results of gene therapy for hemophilia B contrast with those of factor replacement therapies, where lifelong infusions ...

It was found that those with severe hemophilia B have less than 1% of normal factor IX activity, often experiencing spontaneous bleeding that can cause joint damage or life-threatening events.

A single infusion of adeno-associated virus (AAV)-mediated gene therapy led to sustained clinical benefit and no late-onset ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...

In a major breakthrough, UK-based researchers have found that a one-time gene therapy approach for haemophilia B remains safe ...

The approval keeps uniQure in pole position over its closest rival in the haemophilia B gene therapy race, Pfizer/Roche, whose fidanacogene elaparvovec candidate is in the BENEGENE-2 trial, due to ...

In the last of a series, columnist Jennifer Lynne talks with Chip Hailey about how gene therapy has transformed his life with hemophilia B.

According to a new study in Haemophilia, up to 60% of patients with severe HA have no known family history, making early ...

In a 13-year follow-up by St. Jude Children’s Research Hospital and University College London published in The New England ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's ...