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 ...

Subsequent studies by other investigators confirmed those early results, ultimately leading to the FDA approval of ...

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.

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

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 ...

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

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