July 19, 2021
BioMarin announced new data for their investigational gene therapy, valoctocogene roxaparvovec currently in a phase 3 study to treat hemophilia A. Results included positive data for the 134 participants who have been involved in the study for more than a year.
July 9, 2021
Sigilon Therapeutics' cell therapy trial for hemophilia A is on hold due to an FDA hold after a patient developed inhibitors. Three patients have undergone treatment and one developed antibodies to inhibit factor VIII.
July 9, 2021
The US Food and Drug Administration approved ASC Therapeutics' request to open a clinical trial in the US for the safety and efficacy of ASC618, a second-generation gene therapy for hemophilia A. The trial will begin in July 2021 and will test the one time therapy in up to 12 adults.
June 18, 2021
A new study found that those with non-severe hemophilia A were also at risk for developing inhibitors. The findings highlight the importance of closely monitoring these patients in order to prevent or delay the onset of inhibitors.
June 14, 2021
A study published in the journal Haemophilia shows that switching or starting prophylactic treatment Hemlibra may lead to a significant reduction in treatment related expenses. The study found there was about 27.5% drop in expenses over a six month period.
June 9, 2021
The Coalition for Hemophilia B is hosting a contest for those with hemophilia B, their family members, or caregivers. Participants can enter through creating a comic or coloring book and are judged in various age categories. Prizes are $500 for each category and submissions are due by June 30, 2021.
May 25, 2021
Updates from the Phase III HOPE-2 clinical trial of etranacogene dezaparvovec, an investigational gene therapy for hemophilia B patients, were shared at the ASGCT Virtual meeting in May. The data found that AAVs appear to be safe and effective in patients with and without pre-existing neutralizing antibodies.
May 19, 2021
BioMarin releases key findings in gene therapy study of Roctavian, showing no signs of tumors, malignancy, or permanent integration to the DNA of mice.
May 18, 2021
Catalyst Biosciences announced the first dosing of their Marzeptacog Alfa, also known as MarzAA, in a phase 1/2 clinical trial of hemophilia A patients with inhibitors who are being treated with Hemlibra.
May 7, 2021
Severe hemophilia A patients who were treated prophylactically with Jivi showed the treatment was highly effective in preventing bleeds during and after minor surgeries.
April 16, 2021
A first patient has been dosed with Efanesoctocog Alfa, formerly known as BIVV001, in a phase 3 clinical trial evaluating its safety and efficacy in boys with severe hemophilia A.
April 14, 2021
Hemophiliac Jonathan Hill created a graphic novel based on his memoir and characters and adventures he came up with as a way to cope with his bleeding disorder. The graphic novel is available to order free through bloodofthepaladin.com.
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.
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 firstname.lastname@example.org.