Progressive joint disease caused by repeat bleeds, a condition known as hemophilic arthropathy, affects people with moderate hemophilia in Nordic countries regardless of age, commonly causing them pain and restricting their mobility, a study found. Middle-age patients, those 41 to 50 years old, felt their overall quality of life…
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BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene roxaparvovec) in combination with corticosteroids in men with severe hemophilia A. Top-line data from the 52-week (one year) study are expected to be released in early 2023, according to a…
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…
On a recent call with others in the bleeding disorders community, a poignant concern emerged: the fear that the harrowing history of hemophilia‘s dark days may be slipping from our collective memory. The “hemophilia holocaust” remains a haunting chapter in medical history, etching an indelible mark on…
Having a COVID-19 infection had no significant impact on bleeding episodes, nor on the levels of factor replacement therapy used in treatment, for people with hemophilia, a new study from China found. The study did reveal, however, that, compared with adults, children with hemophilia had a significantly lower…
A single infusion of valoctocogene roxaparvovec, BioMarin Pharmaceutical’s investigational gene therapy, can lead to the sustained production of factor VIII (FVIII) in people with hemophilia A, according to a biopsy study that showed the presence of the treatment’s gene constructs in the liver over nearly four…
The European Commission has granted conditional marketing authorization to BioMarin’s one-time gene therapy Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe hemophilia A who have neither inhibitors nor detectable antibodies against adeno-associated virus serotype 5 (AAV5). The decision, which comes a couple of months after a…
Treatment with the extended half-life (EHL) therapies Eloctate (efmoroctocog alfa) and Alprolix (eftrenonacog alfa) was found to safely and effectively control bleeding during major orthopedic surgeries in people with hemophilia A or B, according to real-world data from two Italian centers. The therapies helped patients maintain…