uniQure has treated the first patient in a Phase 2b dose-confirmation study of its investigational gene therapy, AMT-061, for people with severe and moderately severe hemophilia B. The trial is still recruiting participants. AMT-061 uses a viral vector, AAV5, to deliver the gene for a mutated clotting factor…
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When parenting a child with hemophilia, it’s important to understand how you can support them as they learn to live with the bleeding disorder. The two most common types of hemophilia, type A and type B, primarily affect males, while hemophilia C affects both…
The U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information for Altuviiio (efanesoctocog alfa), a long-lasting factor VIII (FVIII) replacement therapy for hemophilia A, to include the full results from a clinical trial, the XTEND-Kids study. As requested by Sanofi, which markets…
Sometimes I hear other women joke about their husbands being their “extra child,” and I can’t help but nod in understanding. Living with Jared, who has severe hemophilia B and a seizure disorder, means I occasionally slip into caregiver mode with him, too. But here’s what might surprise you:…
A genetic mutation for hemophilia has passed through five generations of my family. We can trace it from my great-grandfather, known simply as a “bleeder,” to my nephew. My great-grandfather passed the genetic mutation to my grandmother. My grandmother passed the genetic mutation to my father. My father…
Due to the global COVID-19 pandemic, World Hemophilia Day (WHD) will be a little different this year to keep everyone safe. Still, there are multiple ways to participate in the April 17 event intended to increase awareness and understanding of hemophilia and other bleeding disorders. Begun in 1989 and…
In recognition of World Hemophilia Day (WHD), Catalyst Biosciences is hosting an “Ask Me Anything” (AMA) session on Reddit on April 17. During the Reddit AMA event, which will take place at noon EST Wednesday, clinicians will answer questions from patients and caregivers about bleeding disorders including hemophilia. A…
Enzyre has raised €12 million (over $12 million) to speed the development of EnzyPad, a device that could one day allow people with hemophilia A to monitor their blood-clotting status in real time from anywhere, including their home. “We are delighted to have closed this successful financing…
As part of a new partnership focused on hemophilia treatment and management, members of insurer Highmark will receive medication exclusively from Option Care Health, Soleo Health, and the Hemophilia Center of Western Pennsylvania. The collaboration that begins in January is aimed at keeping down costs associated with…