I love sitting by a campfire. Stories and laughter are shared, s’mores and hot dogs are roasted, and memories are made. Tending the fire to keep the flames burning requires constant attention, and it takes my mind off my troubles. Watching the flames leap and dance is mesmerizing. However, the…
I first met Kim Phelan, chief operating officer of The Coalition for Hemophilia B, in person at a recent symposium in Dallas. From the moment we spoke, I was in awe of her. Despite the hundreds of attendees, she took the time to engage with me personally.
Raising two sons with severe hemophilia A has been a journey filled with ups and downs, joys and fears. I lived in constant fear when my first son, Julian, was a baby. Not only was I concerned about feedings, diaper changes, and making sure he was breathing in…
This past week has been a whirlwind of activity for our family. My husband, Jared, who has severe hemophilia B, experienced a knee bleed that required careful management. But that didn’t stop him from fulfilling his responsibilities around the house and making progress with his budding bottled food business.
Qfitlia (fitusiran) is an RNA-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, with or without inhibitors. It is given via under-the-skin, or subcutaneous, injections that can be self-administered.
Altuviiio (efanesoctocog alfa) is an approved long-lasting factor VIII (FVIII) replacement therapy used to reduce the frequency of bleeding episodes or control and manage active bleeds, including those occurring during surgery, in children and adults with hemophilia A.
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B. This designation is given to therapies that are meant to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. It offers…
Before I became a parent, I never imagined I’d be finding veins and accessing ports. But after my sons were born with severe hemophilia A with inhibitors, my husband and I quickly learned the basics of caregiving, by God’s grace. Over the years, people have said, “Cazandra,…
A European Medicines Agency (EMA) committee has issued a positive opinion recommending conditional marketing authorization of gene therapy Durveqtix (fidanacogene elaparvovec), developed by Pfizer, to treat adults with severe and moderately severe hemophilia B. The European Commission will review the recommendation from the EMA’s Committee for Medicinal…
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