Rixubis (nonacog gamma) for hemophilia
Rixubis (nonacog gamma) is a replacement therapy approved to prevent and treat bleeds, including those occurring during surgery, in adults and children with hemophilia B.
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The joy of lobbying the Senate on behalf of my sons with hemophilia
Last Thursday, I visited Washington, D.C., to join my fellow blood brothers and sisters in lobbying legislators regarding issues that directly affect the bleeding disorders community. I must admit that I was a little nervous. Several times, I wondered, “What in the world am I doing here? I’m only…
Undertaking a pilgrimage of faith in raising 2 sons with hemophilia
There’s a pilgrimage site less than 10 minutes from my home in central New Mexico. That site, El Cerro de Tomé (Tomé Hill), is a natural landmark that’s served El Camino Real travelers for centuries. People journey to the top of the hill, 400 feet above the Rio Grande…
Hemlibra controls hemophilia A bleeds over nearly 5 years: Analysis
Hemlibra (emicizumab-KXWH) safely controlled bleeds among hemophilia A patients without inhibitors over nearly five years of preventive treatment, according to long-term data pooled from two Phase 3 clinical trials. “Five years of follow-up data showed a favorable benefit-risk profile of [Hemlibra] prophylaxis,” the researchers wrote in “…
FDA OKs first-in-human study of F9 gene therapy for hemophilia B
Intellia Therapeutics and Regeneron are planning to begin, by mid-year, the first-in-human trial testing a CRISPR/Cas9-based Factor 9 (F9) gene-editing therapy for people with hemophilia B. The announcement of the planned launch of the Phase 1 clinical trial, in an Intellia financial report, follows the recent approval by…