uniQure/ CSL Behring's Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years
CSL and uniQure's Hemgenix shows sustained efficacy in hemophilia B, reducing bleeding rates by 90% and eliminating ...
CSL Behring confirms long-term efficacy of HEMGENIX in hemophilia B treatment
Global biotechnology leader CSL Behring (ASX:CSL; USOTC: CSLLY (OTC:CSLLY)) announced the four-year results from the HOPE-B ...
MedPage Today5h
Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
UCSD Guardian11d
UCSD offers novel gene therapy for hemophilia B, a rare blood disorder
UC San Diego announced that it is offering a newly Food and Drug Administration-approved gene therapy for hemophilia B, a ...
Pharmaceutical Technology on MSN3d
Italian charity seeks EMA approval for rescued gene therapy
Telethon Foundation is seeking approval for a gene therapy to treat the rare disease Wiskott-Aldrich syndrome.
Gene therapy may be 'one-shot stop' for rare bone disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
NHS to offer 'groundbreaking' sickle cell gene therapy
The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts ...
BioSpace9h
Novo’s Bispecific for Hemophilia Aces Phase III Pediatric Trial
Novo Nordisk’s bispecific antibody Mim8 prevented bleeding events and caused no adverse safety events in the Phase III FRONTIER3 trial. The company plans to file for approval this year, hoping to ...
Hemophilia Treatment Market Valuation is Projected to Reach US$ 21.48 Billion by 2033 | Astute Analytica
Growing advancements in haemophilia treatment underscore a significant shift toward targeted and long-lasting therapeutic ...