My dad’s life was incredibly difficult. He was in pain all the time and misunderstood by others. He grunted and grumbled, and walked with an exaggerated limp. People thought he was a hypochondriac who wouldn’t stop talking about his ailments. Some found him interesting, others thought…
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I gaze at the glorious sunrise as I take off in my red RAV4. On this particular trip I plan to make my way down from my home in Northern California to Los Angeles. My daughter and I will most likely push through the 12+ hour drive in…
Hemophilia A patients with factor VIII (FVIII) inhibitors show deficiencies in regulatory B- and T-cells — immune cells that typically dampen immune and inflammatory responses — but their levels can be restored with successful immune tolerance induction (ITI), a study shows. These findings shed light on how ITI — given…
A single dose of Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for severe hemophilia A from BioMarin Pharmaceutical, maintains factor VIII (FVIII) levels at near-normal or normal levels for up to five years, with most patients staying off prophylaxis, or preventive treatment. These lasting benefits were observed in GENEr8-1…
Recently, my wife, Cazandra, and I entered an extraordinary time — when our adult sons are enjoying a period of peace with their bleeding disorders. Several months passed, and the only health issues that came into focus were dental surgery for Julian, our oldest, and some minor discomfort for…
Tremeau Pharmaceuticals has received guidance from the U.S. Food and Drug Administration (FDA) on how to conduct a pivotal Phase 3 trial exploring rofecoxib (TRM-201) in patients with hemophilia-associated joint pain. The advice was offered during a formal meeting with the agency, where both parties agreed on the design…
Most people with moderate or mild hemophilia A who received preventive treatment with Hemlibra (emicizumab) in the HAVEN 6 clinical trial had no bleeds requiring treatment over a median follow-up of over a year, new data show. The findings were presented at the 30th International Society on Thrombosis and…
Hemophilia has been a gift. Let me explain. I know some people will struggle to understand my viewpoint. I realize that they have suffered great physical and emotional pain because of this bleeding disorder. Some have lost relationships, jobs, and mobility, and have a reduced quality of life. I get…
Obesity increases the frequency of joint bleeds and degree of pain, and lowers overall life quality of people with hemophilia in the United States, most healthcare providers agreed in an online survey. But a sizable number, about 40%, did not discuss weight with patients during care…
A single-center U.S. study found relatively little genetic testing was done for girls with a 50% risk of being a hemophilia carrier — people with the altered gene who don’t have hemophilia themselves but can pass it to their children — over a four-year period in Washington state.