In our Newsroom we share some recent headlines and links to articles that may be of interest to our bleeding and clotting disorder patients, families, and providers.

UniQure Shares Positive Data from AMT-061 Gene Therapy Trial for Hemophilia B

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. 


Study Finds Passive Stretching, Manual Therapy Helps Joints of Hemophilia Patients

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. 


Results Published from Patient Satisfaction Survey Show Positive Reviews of HTCs Nationwide

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.


Data Shows Hemlibra Improves Quality of Life for Children With Hemophilia A

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.


Study Finds Good Long Term Outcomes After Knee Replacements for Hemophilia Patients

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. 


NIH Awards $1.5M Grant to DNA Medicine Institute to Develop Rapid Fingerstick Test for Factor FVIII

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). 


First Participant Dosed in Phase 3 Study for Pfizer and Sangamo Gene Therapy

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. 


CSL Behring States Stimate Will Not Be Resupplied Until 2022

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.


CSL Behring Will Discontinue Mononine for Hemophilia B

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.


Genentech Provides Update on Particles in Hemlibra

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. 


FDA Rejects BioMarin's Gene Therapy for Hemophilia A

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. 


Sanofi's BIVV001 Phase 1/2 Trial Results Show Promise

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.

News Content Disclaimer:

Links to articles listed may take readers to websites separate and out of the control of the CU School of Medicine and the Hemophilia and Thrombosis Center. The CU School of Medicine and the Hemophilia and Thrombosis Center are not responsible for any errors, inaccuracies or opinions that may be expressed in any of the linked articles.

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