Sangamo Therapeutics has handed over the development of SB-525, a gene therapy for hemophilia A, to Pfizer, which will now advance the therapy into Phase 3 clinical trials. Pfizer has already started recruiting participants for the lead-in Phase 3 trial (NCT03587116). which will support…

This month has brought a holiday rush of hemophilia news on potential new treatments, studies, and acquisitions — all of which serve to raise hope during this solstice season, as we move past the shortest day of the year. Solstice can be a…

Takeda has presented new real-world data to support the cost-effectiveness and persistent benefits to patients of its hemophilia A and B therapies: Adynovate [antihemophilic factor (recombinant), PEGylated] and Feiba (anti-inhibitor coagulant complex). The findings were discussed at the recent 61st American Society of Hematology…

With Christmas only a few weeks away, Hemophilia Association of the Philippines for Love and Service (HAPLOS) threw its annual Christmas party. It’s a modest event, but seeing people gather, spread cheer, and enjoy one another’s company is heartwarming. The party is also a time to express gratitude to…

BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A. The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly…

When my daughter was little, she had the most amazing dance teacher, Jenna. Jenna was a mom of boys who loved the rough and tumble, but her dance studio was all about learning to move young bodies with grace and fluidity. While we no longer head off to Jenna’s classes…

For the longest time, my son Caeleb being in a wheelchair was just part of the normal workings in my home. The constant bleeds that kept him immobile were in the forefront of my thoughts as I watched him struggle to get around. But after five years without bleeds,…

I attended the Hope for Hemophilia conference last weekend and met some wonderful people. I delivered a keynote address and shared my story of raising sons with hemophilia and holding on to hope. It was a special experience and I am grateful for the opportunity. The day after…

I recently attended a national conference for women with hemophilia. Common themes emerged as I listened to women’s stories of life with bleeding disorders. The number of women dismissed by top medical professionals working for hemophilia treatment centers (HTCs) was alarming. I wondered why this is happening. Dominant…

You’ve been talking to prospective Mr. Wonderful for a few weeks now. You have a lot in common, and if he looks anything like his profile picture, you know he’s going to be some significant eye candy. You have started to flirt through email. You might even be…