SPK-8016 for hemophilia A
SPK-8016, also known as RG6358, is an experimental gene therapy that was being investigated for hemophilia A patients who had developed neutralizing antibodies (inhibitors) against clotting factor VIII (FVIII).
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AMT-060 for hemophilia
AMT-060 was a first-generation gene therapy for hemophilia B that was tested in clinical trials as a potential treatment for reducing the risk of bleeding episodes in patients.
Monthslong shortage forecast for Rebinyn 3,000 IU for hemophilia B
Unexpectedly high demand for the 3,000 international units (IU) vial size of Rebinyn (nonacog beta pegol) has resulted in a temporary shortage of the Novo Nordisk factor IX (FIX) replacement therapy for those with hemophilia B, according to the company. In a statement, Novo Nordisk said…
A new initiative seeks to preserve the painful history of hemophilia
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…
Helping my son stand when hemophilia knocks him off balance
Today started out like most any other day. I walked into the room of my youngest son, Caeleb, to wake him up to get ready for school. I love the ritual of turning on the lights and singing a familiar tune to my family. As I began, however, I found…
Pain and mobility issues common with moderate hemophilia: Study
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…
PEGylation for hemophilia
PEGylation is one of the molecular technologies that has been used to create extended half-life replacement therapies for hemophilia. These longer-lasting therapies don’t need to be administered as often, reducing the treatment burden for patients.
‘The Hub’ aims for hemophilia B awareness, empowerment
As my beloved hematologist pointed out during my last hemophilia clinic appointment, my health situation is rare. I have at least two identified bleeding disorders: von Willebrand disease and hemophilia B. I’ve met others along my journey who have two disorders, too, but the situation…
Lightening the load is crucial when parenting a son with hemophilia
I have battled weight and self-image issues since I was a teenager. Being present with my hospice patients day in and day out becomes draining. I take on any pain and disappointment my husband and sons experience. And when I witness my boys struggle because of hemophilia, the…
Cyklokapron (tranexamic acid) for hemophilia
Cyklokapron (tranexamic acid) is an approved hemophilia medication that's designed to control or prevent bleeding episodes in patients, and reduce the need for replacement therapy during and following a tooth extraction.