Officials of the U.S. Food and Drug Administration and Alnylam Pharmaceuticals have agreed on safety measures and a “risk mitigation strategy” to allow the resumption of clinical studies of fitusiran, the company said in a recent press release. All ongoing trials of fitusiran had been suspended after the Sept. 7 death…

Last week, I was on Facebook and a post I had been dreaming about appeared on my timeline. The U.S. Food and Drug Administration has approved a new therapy, Hemlibra (emicizumab-kxwh), for patients with hemophilia A and inhibitors. It’s the first new medicine for inhibitors in almost…

The advancements in modern medicine have meant that people with hemophilia are living longer than ever, however, this brings its own set of problems for the medical profession. People living with the disease are just as likely to have other health issues when they get older as those without hemophilia,…

The European Medicines Agency has recommended marketing authorization for Shire’s Adynovi (BAX-855) as a treatment for adults and adolescents with hemophilia A. Approval would mean the therapy could be used twice a week to prevent bleeding episodes and to counter bleeding when episodes do occur. The U.S. Food and Drug Administration…

The U.S. Food and Drug Administration (FDA) has approved Hemlibra (emicizumab-kxwh) for routine prophylaxis to prevent or lessen bleeding episodes in children and adults with hemophilia A with factor VIII inhibitors, Genentech announced. Hemlibra is first newly approved treatment for this patient group in almost 20 years, the…

When young Max Levy had to have a port put in his chest for hemophilia medication, his family nicknamed him Iron Man after one of his favorite Marvel action heroes. The story touched a lot of hearts and was even shared by Robert Downey Jr. on his…

One of the major hurdles people with hemophilia face is complying with their medication schedule — in particular, injecting themselves several times a week with clotting factor. MORE: Seven recommended books to learn more about hemophilia Clotting factor has a relatively short half-life (the time it takes for a person’s…

An ongoing Phase 1/2 clinical trial evaluating a gene therapy, SPK-9001, in people with hemophilia B is expanding its enrollment by up to five more patients, who will be given a new version of the therapy, its developer announced. One patient in this group has already received SPK-9001 produced under…

Swedish Orphan Biovitrum (Sobi) announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial. The Phase 4 study (NCT03103542), sponsored in collaboration with Bioverativ Therapeutics, is actively recruiting participants who have developed inhibitors and who have failed to respond to other therapies. Researchers will…

This children’s video from Centers for Disease Control and Prevention focuses on how to tell your friends that you have hemophilia. MORE: How kids can play safe with hemophilia Billy, a young boy with hemophilia, answers some of the questions that his friends ask about the disease. He…