News

Hemophilia B gene therapy demonstrates long-term success
A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...
MedPage Today on MSN7d
Hemophilia B Gene Therapy Sustains Efficacy Over a Decade Later
Subsequent studies by other investigators confirmed those early results, ultimately leading to the FDA approval of ...
Nature2d
One dose of gene therapy gives years of relief from blood disorder
Gene therapy has proved to be a safe long-term treatment for haemophilia B. The benefits of a single dose persist for at ...
Managed Healthcare Executive1d
Gene Therapy Cuts Bleeding and Medicine for Hemophilia B Patients for Over a Decade
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.
Technology Networks6d
Gene Therapy for Hemophilia B Demonstrates Long-Term Success
In a 13-year follow-up by St. Jude Children’s Research Hospital and University College London published in The New England ...
First NHS patient given world's most expensive drug that costs £2.6 million per dose
A UK man became the first NHS patient to receive a multimillion pound wonder drug, Hemgenix, to help treat the clotting ...
Daijiworld6d
Gene therapy offers long-term relief for Haemophilia B: Study
In a major breakthrough, UK-based researchers have found that a one-time gene therapy approach for haemophilia B remains safe ...
The New England Journal of Medicine8d
Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B
These data support the long-term efficacy and safety of AAV gene therapy for severe hemophilia B. (Funded by the U.K. Medical Research Council and others; ClinicalTrials.gov number, NCT00979238 ...
Managed Healthcare Executive6d
Half of Severe Hemophilia A Cases Have No Family History, Delaying Diagnosis but Not Raising Inhibitor Risk
According to a new study in Haemophilia, up to 60% of patients with severe HA have no known family history, making early ...
pharmaphorum3d
Pfizer's haemophilia B gene therapy meets phase 3 primary endpoint
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 ...