Shire has launched a Phase 3 clinical trial (NCT02585960) with Adynovate, a drug approved to help treat and control bleeding in children and adults with hemophilia A. The trial is currently recruiting participants. Adynovate is an injectable recombinant factor VIII (FVIII), designed using pegylation technology, which allows it to last…
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Living with pain is exhausting. However, those living with pain have numerous ways to manage their condition, seeking relief through medication, physical therapy, massage, chiropractic care, and even hypnosis. Knowing the cause of that pain is helpful for doctors and patients as they work to find ways to cope and…
The parents of most children with severe hemophilia A who took part in the XTEND-Kids clinical trial of Altuviiio (efanesoctocog alfa) reported in exit interviews that the once-weekly treatment led to notable improvements in mood for their sons, with some reporting a greater ability for their boys to…
The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval of the gene therapy Roctavian (valoctocogene roxaparvovec) to treat adults with severe hemophilia A. The FDA is now expected to decide by the end of March 2023 on whether…
Eptacog beta was safe and 100% effective at preventing bleeds during and after minor surgeries in hemophilia A patients with inhibitors, according to data from a Phase 3 trial. The treatment’s efficacy at managing bleeds dropped with major surgeries, with post-operative success seen in 66.7% of evaluated cases. Overall, these…
I’ve been struggling with hip bleeds lately. I’m in the middle of recovering from a bleed on my right hip, which may have started because I put too much pressure on it too soon. I’m frustrated that I’ve been bedridden so often these past few months. It’s hard…
My local hemophilia organization just held its annual Christmas party. During these celebrations, we are reminded that we belong to a community that empowers, cares, and provides us with support and service. Christmas is a time for giving, showing gratitude, and spreading love. During our Christmas party, it was heartwarming…
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My First Steps into Parenthood
As I write this, I’m bedridden from a bleed in my right hip. I’m learning what it means to have both hemophilia and a baby girl. Although I could see this bleed as a spirit-breaker, I’m readjusting, and I’ll be better prepared for episodes like this in the future.
The U.S. Food and Drug Administration (FDA) has scheduled its inspection of the facility in California where BioMarin Pharmaceutical would produce the gene therapy Roctavian (valoctocogene roxaparvovec) if it’s approved. The agency is reviewing a biologics license application (BLA) from BioMarin that seeks approval of Roctavian to…
The presence of inhibitors and excessive body weight increase the sense of disease burden felt by parents of children with moderate to severe hemophilia, a study reports. The study “Burden on parents of children with hemophilia: the impact of socio- demographic and child’s medical condition” was published in…