The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval of the gene therapy Roctavian (valoctocogene roxaparvovec) to treat adults with severe hemophilia A. The FDA is now expected to decide by the end of March 2023 on whether…

Be Biopharma has raised $92 million in series C financing to support the first-in-human Phase 1/2 trial of BE-101, the company’s B-cell therapy candidate for hemophilia B. Proceeds from the funding round will be partly used to generate proof-of concept data from the BeCoMe-9 trial (NCT06611436), which…

Recently, I attended the Florida Bleeding Disorders Association’s Women’s Educational Retreat in Hollywood, Florida. I came away with several important takeaways, but one presentation by Lucila Ramirez Donaldson of Genentech truly resonated: caregiver burnout. Many of the women attending were mothers or partners of people living with hemophilia. I’ve…

When I tell people I’m married to someone with hemophilia, I get different reactions. Some people are genuinely curious and want to know more about my husband’s condition. They’ll ask several questions about the hows and whys of his bleeds, and are often curious about treatment. On the other hand,…

In 2000, actor Tom Hanks starred in the movie “Cast Away.” His character, Chuck Noland, traveled the world as a systems analyst for FedEx, and his plane crashed in the South Pacific. He was on a deserted island, and his only connections with the world were the FedEx…

Eating well is important for everyone, but particularly for those living with hemophilia. Eating a diet that encourages strong bones and muscles and helps keep you at a healthy body weight is essential. MORE: Young boys explain what it’s like to live with hemophilia Obesity is becoming a major problem when it…

Eating well is important for everyone, but particularly for those living with hemophilia. Eating a diet that encourages strong bones and muscles and helps keep you at a healthy body weight is essential. MORE: Young boys explain what it’s like to live with hemophilia Obesity is becoming a major problem when it…

Hemophilia patients may now benefit from financial assistance for out-of-pocket expenses, health insurance premiums, and incidental medical costs following the launch of a new program from The Assistance Fund (TAF). Founded in 2009, TAF is an independent nonprofit aimed at helping eligible patients and their families cover costs not…

Successful immune tolerance induction (ITI) — a type of treatment that aims to re-educate the immune system to prevent the formation of inhibitors, or neutralizing antibodies targeting clotting factors — was associated with lower treatment costs in hemophilia A patients with inhibitors, a study in Brazil reported. Conversely, treatment…