The U.S. Food and Drug Administration (FDA) has approved ASC Therapeutics’ request to open a clinical trial in the U.S. into the safety and early efficacy of ASC618, its second-generation gene therapy for hemophilia A. A Phase 1/2 trial (NCT04676048), set to start this month, will test…
Search results for:
An experimental form of genome editing called clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a faster and cheaper method of treating people with hemophilia, compared to the current DNA editing method. Because hemophilia is due to DNA mutations, the genome editing could be used to efficiently slice at the DNA,…
Note: This column describes the author’s own experiences with surgical procedures. Not everyone will have the same response to these operations. Consult your doctor about the potential risks and benefits of any procedure you’re considering. As I get older, the thought of needing any kind of surgery scares the…
Joint problems caused by chronic bleeding often trouble people with hemophilia, especially in the knees, ankles, and elbows. A paper by scientists in Spain and the U.S. reviewed existing literature on knee treatments for people with this disease, focusing on best current practices — including a preventive, if inconveniently lifelong, therapy — and future…
Four years ago, I relocated to a rural community. My hemophilia treatment center (HTC) was six hours away, so I met with a local hematologist to come up with a plan for treatment. I am a woman. I was speaking to a female physician who specialized…
Adynovate (antihemophilic factor, rurioctocog alfa pegol), sold as Adynovi in Europe, is a recombinant (lab-made) clotting factor VIII (FVIII) concentrate given as an intravenous (into-the-vein) injection to prevent and control bleeding in children and adults with hemophilia A.
Adults with hemophilia who switched from standard to extended half-life replacement treatment needed fewer infusions, had fewer bleeds per year, and adhered better to their treatment schedules, a Finnish study shows. Switching to extended half-life replacement treatment increased the mean yearly costs by nearly €19,000 (about $22,000) for…
Speaking at the Hemophilia Federation of America’s Mild Matters Summit last year, former Hemophilia News Today columnist Shellye Horowitz shared how her treatment plan changed after she researched the genetic variant that caused her hemophilia A. Although she’s classified as “mild,” her factor treatment…
In the past, no one believed women could have hemophilia. The medical community assumed that one X chromosome would compensate for the other. Doctors now know about the process of duplicate X chromosome inactivation, called “lyonization.” As the inactivation process is random, some female…
Hympavzi (marstacimab-hncq) is an antibody-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, without inhibitors. It is given as weekly subcutaneous, or under-the-skin, injections that can be self-administered.