Antifibrinolytics are medicines that promote blood clotting by preventing or slowing down a process called fibrinolysis, which is the break down of blood clots. Antifibrinolytics are used as a treatment for hemophilia, in surgical procedures to prevent excessive blood loss, and for heavy menstrual bleeding. How antifibrinolytics work…
Hemophilia is a blood disorder that is characterized by a shortage of specific clotting factors that help to form blood clots and stop bleeding. The primary treatment for hemophilia is replacement therapy that supplies the clotting factors to hemophilia patients who lack them. Other approved hemophilia treatments include…
Hemophilia is a bleeding disorder caused by a mutation in genes that provide instructions for blood clotting factors — the proteins that help in blood clotting. Mutations in the genes for clotting factors VIII, IX, and XI result in their deficiency, causing hemophilia A, hemophilia B, and hemophilia C, respectively.
Gene therapy is an experimental treatment technique that uses genes or genetic material to treat or prevent disease. Human clinical trials are underway to test potential gene therapies for hemophilia. What is hemophilia? Hemophilia is a genetic blood clotting disorder where patients do not make enough of the factors that…
SB-FIX, developed by Sangamo Therapeutics, is an investigative gene therapy for hemophilia B that, by making changes to the DNA sequence, may allow hemophilia B patients to make their own factor IX protein. (Factor IX is missing or present at low levels only in patients with hemophilia B,…
Mother’s Day is this Sunday. The day dedicated to honoring mothers began in the early 1900s. It’s a special time of joy and celebration, with families going out for lunch and mothers receiving cards and flowers. That’s the picture-perfect version of what Mother’s Day looks like in a television…
Brandi’s journey with hemophilia Recently, I had the opportunity to speak with Brandi Worthington about her experiences as a woman living with hemophilia. Brandi was in her 30s when she received a diagnosis that linked hemophilia to her long-standing bleeding issues. This revelation came 23 years after the onset of…
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…
Having a family member admitted to the hospital throws the entire family’s routine into absolute chaos. We lose our control of time, as well as our privacy. While it is great to see a friend or to hear good news from the medical staff, we are unable to dictate…
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.
Get regular updates to your inbox.
