November 20, 2020
A Phase 3 Trial for AMT-061, a gene therapy treatment for hemophilia B being developed by uniQure, showed increased factor IX activity and controlled bleeding while markedly reducing the need for other treatments.
November 11, 2020
A small study, published in Haemophilia recently, shows that patients who did passive stretching and also received manual therapy, reduced problems with joint bleeds.
November 3, 2020
A manuscript reviewing research from a survey of U.S. bleeding disorder patients who seek care from an HTC was published in Haemophilia outlining the positive reviews of their care.
October 26, 2020
A study shows that preventative treatment with Hemlibra (emicizumab) improves health related quality of life in children with hemophilia A who develop inhibitors.
October 23, 2020
A new, single-center study from South Korea found that most people with hemophilia who undergo knee replacement surgery have favorable outcomes.
October 12, 2020
The DNA Medicine Institute was awarded a $1.5 million grand from the National Institutes of Health to develop a rapid, point-of-care fingerstick blood-based test for Factor FVIII and Roche's hemophilia A treatment emicizumab (Hemlibra).
Oct 7, 2020
Pfizer and Sangamo announced that the first participant has been dosed in their Phase 3 AFFINE study of giroctocogene fitelparvovec (SB-525) in an investigational gene therapy for hemophilia A patients.
October 2, 2020
CSL Behring issued a statement regarding Stimate, which was involved in a voluntary recall. They announced that Stimate will not be resupplied before 2022.
September 21, 2020
CSL Behring has announced they will discontinue the manufacturing of Mononine to treat hemophilia B patients. The factor medication has been on the market since 1992 but advances in other medications has led to a significant drop in demand from patients. Mononine will be available through mid-2021 giving patients who do use the therapy to adjust to new options on the market.
September 17, 2020
Roche and Genentech have provided an update to the measures they are applying to reduce particles that were identified in Hemlibra doses in 2019. To date the particles have shown no risk or impact to patients and the manufacturer is working on ways to reduce or eliminate them. Genentech has proposed use of a transfer needle with a filter to provide additional confidence for Hemlibra's use.
August 19, 2020
In a surprising decision, the FDA refused to approve BioMarin's gene therapy valoctocogene roxaparvovec, also known as valrox. The decision requires BioMarin to finish their phase 3 trial and complete two years of follow up data to show the treatment's durability and efficacy.
September 17, 2020
Sanofi announced results from their phase 1/2a EXTEN-A trial for their investigational therapy BIVV001 to treat patients with hemophilia B. The trial showed patients achieving a FVIII half-life of 43 hours, a more than three fold increase from conventional therapy. The results appear to show that use of this treatment could reduce severe cases to behave more like a mild case. No detection of inhibitor development or adverse events were reported in the study.
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