Hemophilia News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to hemophilia throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read stories of 2019. No.
Search results for:
Switching from on-demand treatment to regular preventive therapy with extended half-life products may mean fewer bleeds, healthier joints, and better quality of life for people with hemophilia. That’s according to a new analysis of Phase 3 study data from trials involving more than 100 hemophilia patients that spanned a…
Therapeutic plasma exchange (TPE) was found to reduce bleeding and ease disease symptoms in six people with acquired hemophilia A (AHA), a case series reported. These findings support TPE — in which the liquid part of blood is replaced with substitute plasma — as an alternative treatment option in…
A new delivery mechanism of mRNA therapy was found to efficiently reduce hemophilia B symptoms by correcting the protein deficiency that characterizes the disease in a mouse study. This potential new method might be a viable alternative for many clotting disorders. The study, “Systemic delivery of Factor IX messenger RNA…
The European Commission has conditionally approved Hemgenix (etranacogene dezaparvovec), a one-time gene therapy to treat adults with hemophilia B. Hemgenix, originally developed by uniQure, is approved for adults with severe and moderately severe hemophilia B without a history of inhibitors. It’s the first gene therapy for…
Gene therapy has long been touted as a revolutionary hope for people with hemophilia B, promising a one-time treatment to drastically reduce — or even eliminate — the need for frequent infusions. Yet that hope has hit another roadblock. Pfizer announced last week that it’s halting development of…
Note: This story was updated May 8, 2023, to remove hemophilia as one of the specific indications the collaboration will focus on, as these have not yet been publicly disclosed. AstraZeneca and Sernova are teaming up for a preclinical research project that aims to develop new cell therapies…
Intellia Therapeutics and Regeneron are planning to begin, by mid-year, the first-in-human trial testing a CRISPR/Cas9-based Factor 9 (F9) gene-editing therapy for people with hemophilia B. The announcement of the planned launch of the Phase 1 clinical trial, in an Intellia financial report, follows the recent approval by…
As gene therapy is relatively new treatment option for hemophilia B, it's normal to have some questions about it. Here are answers to some commonly asked questions about gene therapy for hemophilia B.