Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…
Search results for:
The Medicines and Healthcare Products Regulatory Agency (MHRA) for the U.K. has approved the resumption of patient enrollment in BioMarin Pharmaceuticals’ Phase 1/2 clinical trial evaluating the investigational gene therapy BMN 270 as a treatment for severe hemophilia A. BioMarin suspended patient dosing in the trial in June because of increasing levels…
Hemophilia treatment can be costly, but understanding insurance options and financial assistance programs may help reduce the burden. This resource highlights key costs and support tools to help you better manage care expenses.
My Life, Our Future (MLOF), a national program founded by leaders in the hemophilia and blood disorder community, marked Feb. 28 — Rare Disease Day — by launching the world’s largest research repository of its kind to researchers and scholars. The program is opening to U.S. scientists and will expand to worldwide scientists in 2018.
Social Clips
Why Hemophilia Is Called ‘A Royal Disease’
Hemophilia is a rare blood condition where people do not have the clotting factor which enables their blood to clot when bleeding. It’s an inherited disease that’s usually passed from mother to son. It’s also a disease that’s been prevalent in European royal families. MORE:Â A brief history of hemophilia…
Albumin fusion is a molecular technology that has been used to create replacement therapies for hemophilia with an extended half-life — treatments that are more long-lasting in the body and can therefore be administered less frequently.
Cyklokapron (tranexamic acid) is an approved hemophilia medication that's designed to control or prevent bleeding episodes in patients, and reduce the need for replacement therapy during and following a tooth extraction.