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.
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Study Shows Esperoct Highly Effective as Prophylaxis Treatment for Hemophilia A

January 29, 2021

An analysis published in the journal ClinicoEconomics and Outcomes Research evaluated the efficacy of Esperoct in preventing bleeds in previously treated patients with severe hemophilia A. Findings showed Esperoct's use as prophylaxis resulted in the fewest traumatic, spontaneous, and joint bleeds of four EHL treatments.

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BioMarin Shares Positive Data for valactocogene roxaparvovec, Gene Therapy

July 25, 2021

BioMarin announced positive updates from their ongoing Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for those with severe hemophilia A. The trial includes participants who have been observed for at least one year post treatment.

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CSL Behring Issues Voluntary Recall of Mononine

January 21, 2021

CSL Behring issued a voluntary recall of one batch of Mononine Coagulation Factor IX (Human), a plasma-derived product used to treat hemophilia B. Risk to patients is considered low. Contact your pharmacy if you have questions about this recall.

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Study Suggests Eloctate Induces Faster Immune Tolerance than other Regimens

January 18, 2021

A study published in the journal Haemophilia shared that Eloctate can induce immune tolerance in people with Hemophilia A more quickly than other regimens. Immune tolerance induction (ITI) is the only strategy known to be effective for removing inhibitors.

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Phase 3 Trial Shows Sustained Drop in Bleeding Rates for Roctavian, Gene Therapy for Hemophilia A

January 13, 2021

Roctavian, an investigational gene therapy for hemophilia A patients, significantly reduced bleeding rates and the need for other treatments over a year according to data from a Phase 3 clinical trail. 

Corona Virus

NHF Shares Information about COVID-19 Vaccines

December 28, 2020

The National Hemophilia Foundation shared a FAQ page with a wealth of information about the new vaccines available for COVID-19. The page addresses many questions people may have, especially focused on those with bleeding disorders, as well as resources for information about the vaccine.

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FDA Puts Clinical Hold on uniQure's Hope B Trial

December 20, 2020

The U.S. Food and Drug Administration announced a clinical hold on uniQure's Phase 3 Hope-B Trial of AMT-061 in severe or moderately severe hemophilia B patients. The hold follows a safety report relating to a possibly related serious adverse event associated with a preliminary diagnosis of HCC, a form of liver cancer in one patient. 

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Updated Phase 1/2 Hemophilia Gene Therapy Study Data Presented at ASH

December 8, 2020

Pfizer and Sangamo announced updated data for their phase 1/2 Alta study of giroctocogene fitelparvovec, an investigational gene therapy for patients with severe hemophilia A. The data was announced at the 62nd Annual American Society of Hematology meeting.

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MarzAA for Hemophilia A or B with Inhibitors Given Fast Track Status from FDA

December 7, 2020

The US FDA granted fast track designation for marzeptacog alfa activated (MarzAA), a lab made version of clotting factor VIIa for the subcutaneous treatment of episodic bleeds in hemophilia A or B patients with inhibitors. MarzAA is moving into a Phase 3 trial.

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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. 

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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. 

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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.

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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Previous articles featured on our website can be found at our archive page here. Please note that links in our archive files may take readers to websites that may have changed their link. In some cases these links do not work anymore. We periodically check and remove articles that have broken links. If you have questions about these articles, please contact us at hemophilia@ucdenver.edu.