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

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

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

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 ...

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

The purpose of this gene therapy is for those people living with haemophilia B, once treated, to be able to produce FIX via the treatment (a one-time treatment), rather than having to receive ...

CSL Behring has added another major European market to the list of those providing reimbursement coverage of its haemophilia B gene therapy Hemgenix, after agreeing an access deal in Germany.

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 ...