The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Phase 3 trial evaluating SB-525 (giroctocogene fitelparvovec), an investigational gene therapy for hemophilia A. This pause in study recruitment and dosing was taken to give the agency time to review changes to the AFFINE…
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The National Hemophilia Foundation (NHF) has opened a grant program to support the local groups and chapters that work with bleeding disorder families, and have watched as their revenue sources dry up during the COVID-19 pandemic. Called the COVID-19 Fund Bridge Grant program, the effort is open to…
A recent survey has shown that the use of appropriate standardized outcome measures of hemophiliac patient treatments in routine clinical practice are not the norm. But implementing these measures could promote more effective personalized therapeutic approaches and reduce treatment-related costs. Hemophilia is a complex disease with a major impact on the…
The relapse rate is higher in older patients with acquired hemophilia A (AHA) treated with immunosuppressive therapies, but it doesn’t affect their overall survival compared with those who don’t relapse, a study suggests. The study, “Relapse pattern and long‐term outcomes in subjects with acquired haemophilia A,” was published in…
Daratumumab — a cancer therapy marketed as Darzalex — can reduce levels of factor VIII (FVIII) inhibitors in people with hemophilia type A, researchers say. Their findings were published in a letter to the editor, titled “Daratumumab rapidly reduces high‐titre factor VIII inhibitors in…
In kindergarten, my teacher had a daily ritual: story time. My friends and I would race to the corner and sit “crisscross applesauce” on the boldly colored carpet, eagerly awaiting the story. Next to recess, it was the best part of the day. The teacher read with excitement, allowing us…
Nothing makes me happier than when my oldest son, Julian, is home and my family is all under the same roof. We cook, laugh, and watch movies. My happy place is with my husband and two sons. One night over dinner, our conversation moved toward the high school band…
Last week, I stumbled upon the Tour de France while aimlessly flipping through TV channels. This annual professional bicycle race is highly esteemed and considered one of the most difficult events in the sport. Watching it was thrilling. The competition was captivating, and the incredible athleticism of the…
When my oldest son was 2 years old, he came up to me with a serious expression and said, “Dad, this monster slapped me right in the face.” I told him I had some monster spray in my room and would ensure that his room no longer contained any strange-looking…
Every family member has a role. Parents usually assume the roles of providers and nurturers, making sure their children have a roof over their heads, food to eat, and plenty of love. Siblings each bring their unique personalities to the home. Some are playful and fun-loving, while others may be…