SPK-8016 for hemophilia A
SPK-8016, also known as RG6358, is an experimental gene therapy that was being investigated for hemophilia A patients who had developed neutralizing antibodies (inhibitors) against clotting factor VIII (FVIII).
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AMT-060 for hemophilia
AMT-060 was a first-generation gene therapy for hemophilia B that was tested in clinical trials as a potential treatment for reducing the risk of bleeding episodes in patients.
Monthslong shortage forecast for Rebinyn 3,000 IU for hemophilia B
Unexpectedly high demand for the 3,000 international units (IU) vial size of Rebinyn (nonacog beta pegol) has resulted in a temporary shortage of the Novo Nordisk factor IX (FIX) replacement therapy for those with hemophilia B, according to the company. In a statement, Novo Nordisk said…
PEGylation for hemophilia
PEGylation is one of the molecular technologies that has been used to create extended half-life replacement therapies for hemophilia. These longer-lasting therapies don’t need to be administered as often, reducing the treatment burden for patients.
Approved Roctavian ‘liberating’ for 1st US patient treated outside trials
In the final days of 2023, an adult with hemophilia A named Maxwell became the first person in the U.S. to receive treatment with the one-time gene therapy Roctavian (valoctocogene roxaparvovec-rvox) — approved in June of last year — outside of clinical trials. “Receiving this treatment is liberating…
Giroctocogene fitelparvovec for hemophilia
Giroctocogene fitelparvovec, formerly known as SB-525 or PF-07055480, is a gene therapy being developed to reduce the risk of bleeding episodes in people with hemophilia A.
Albumin fusion for 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.
Can women have hemophilia?
Although hemophilia, a bleeding disorder that interferes with blood clotting, is more common in men, women can have it, too.
Top 10 hemophilia stories of 2023
Hemophilia News Today brought you coverage of the latest developments in research and advances in treatments for hemophilia in 2023. Here we’ve compiled a list of the top 10 most-read articles published on our site last year. We hope to continue being a resource for the hemophilia community…
Healthcare costs similar for patients on Adynovate, Eloctate: Study
Healthcare costs are comparable for male patients with hemophilia A who are treated with either Adynovate (rurioctocog alfa pegol) or Eloctate (efmoroctocog alfa), according to a real-world study in the U.S. “Results from this analysis may be used to inform product coverage and treatment decisions valuable for both…