A gene therapy being developed by ASC Therapeutics restored levels of the clotting factor missing in hemophilia A in a mouse model of the disease, and further tests in primates and human cells suggest the therapy might also work for people, the company announced. Results from…
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Tampa General Hospital’s cancer institute is the first healthcare center in Florida, and the third in the U.S., to successfully administer the first and only hemophilia B gene therapy, Hemgenix (etranacogene dezaparvovec), to a patient. “The infusion of this therapy is a hallmark of the TGH Cancer Institute’s…
March is Bleeding Disorders Awareness Month (BDAM) and patients, caregivers, advocates, and other supporters are coming together to call attention to conditions like hemophilia and von Willebrand disease that impact more than 3 million people in the U.S. Bleeding disorders are marked by uncontrolled bleeding that occurs from a…
A new year is always filled with possibilities and hope. After living through 2020, most people are looking forward to what is yet to come. While I am not one to make resolutions, there is one thing I will be working on this year. It does not involve me,…
The new year isn’t exactly kicking off on a high note for me. At the end of last year, I headed to Schaumburg, Illinois, to spend Christmas with my family only for my dental crown to crack in half during the trip and decide to make…
COVID-19 has upended our lives. It is hard. As an individual with a chronic condition, I find myself worrying about how it will affect me in ways that may be different from those of my neighbors or co-workers without chronic conditions. Thankfully, the hemophilia community has…
My husband, Jared, and I were recently talking about Rare Disease Day, which falls on Feb. 28. As a person with hemophilia, Jared has faith in its purpose of raising awareness about rare health conditions and the people who live with them. Having dabbled in hemophilia and mental health…
As I write this, it’s Nov. 1 and we’re observing All Saints’ Day. On this day, I remember my mom, who passed away last June. Her final resting place is many islands away from where I live, so I am unable to pay her a visit. Nevertheless, I offer…
A recall on Ferring Pharmaceuticals‘ intranasal desmopressin compounds — used to treat mild to moderate hemophilia A and von Willebrand disease type 1 — is expected to affect the availability of these products until at least mid- to late-2021, according to an advisory from the…
Regeneron Pharmaceuticals and Intellia Therapeutics are expanding their collaboration to develop hemophilia A and B treatments using the CRISPR/Cas9 gene editing technology. The two companies signed a six-year agreement in 2016 to develop, license, and commercialize gene editing-based therapies. “We’re pleased to expand our work…