The Summer Olympics are around the corner. Gymnasts there will soar through the air with grace and precision, showcasing incredible feats of strength and flexibility. Athletes will push their limits, running at record speeds, demonstrating the peak of human endurance. Divers will climb to dizzying heights, performing acrobatic feats as…
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Key leaders in hemophilia therapy development, representing large pharmaceutical and biotech firms as well as academia and contract research organizations, will gather at the Hemophilia Drug Development conference in Boston on Aug. 14 through 16. The meeting is designed to be an industry-dedicated networking and discussion forum, and many of…
I cannot tell you how many times I’ve heard the term “Bubble Wrap.” A child with hemophilia is born: “How am I going to keep this kid Bubble Wrapped and safe?” A kid with hemophilia falls on the playground and bleeds: “I wish we could Bubble Wrap you…
Most people with hemophilia who undergo knee replacement surgery have favorable outcomes, including less pain and improved function, a new single-center study from South Korea reports. The study, “Mid-to Long-Term Survival of Total Knee Arthroplasty in Hemophilic Arthropathy,” was published in the Journal of…
Sangamo BioSciences announced that its zinc finger nuclease (ZFN)-mediated genome editing product, SB-FIX, has received orphan drug designation from the U.S. Food and Drug Administration (FDA), speeding its development as a potential treatment for hemophilia B. Sangamo plans to initiate a Phase 1/2 clinical trial, SB-FIX-1501, in adults with this disease later…
I get many opportunities to travel across the country to speak with families affected by bleeding disorders. Everywhere I go, I see familiar faces — faces that bring me great joy. There are lots of hugs, and each smile warms my heart. My talks change from event to…
Hemophilia B and experimental gene therapy have been in the news lately. I was even surprised to hear a story on the topic while listening to my local NPR station. Hemophilia B is the less common form of the disorder that arises when a…
My grandmother always said, “If you don’t have anything good to say, don’t say anything at all.” She lived by those words, and in all of my memories, I can’t recall her ever speaking ill of anyone. Last week I found myself with nothing good to say, which left my…
Two common themes that appear in my columns are conflict and reconciliation. Once a problem has been resolved and I can reflect on it, I look for insights about how to face the struggles of living with hemophilia. Taking the time to do this has resulted in a change in…
Health Canada has approved fidanacogene elaparvovec, formerly known as SPK-9001 and now marketed under the brand name Beqvez, for adults with moderately severe to severe hemophilia B. Developed by Pfizer, the gene therapy is under review by regulatory agencies in the U.S. and Europe. “Pfizer has…