Although hemophilia treatments are generally effective, women and girls with this disease have specific medical challenges, such as excessive menstrual bleeding, that must be addressed for better clinical care. A study evaluated the symptoms, bleeding management, and clinical outcomes related to menstruation, childbirth, dental procedures, surgeries, and other bleeding events among female…
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CSL Behring has issued a voluntary, pharmacy level recall covering one batch of Mononine, its long-standing treatment for preventing and controlling bleedings in people with hemophilia B that is soon to be discontinued. According to the company’s statement, the recall is a precautionary measure due to…
Rixubis safely and effectively prevents and manages bleeding in children and adults with hemophilia B, a real-world study from South Korea reports. The study, “Safety and effectiveness of Rixubis in patients with hemophilia B: a real-world, prospective, postmarketing surveillance study in South Korea,” was published in the journal Blood…
People with severe hemophilia B are likely to develop hemophilic arthropathy — a painful and degenerative joint disease caused by recurrent bleedings — despite the lower bleeding frequency relative to hemophilia A, a natural history study shows. The data also highlights that prophylactic (preventive) treatment guidelines are not being…
It was Christmas in 1978. My brother was thrilled that Santa had brought him exactly what he wanted — a large, bright red firetruck with a ladder that really worked! (Never mind that we were Jewish. Santa always seemed to make an appearance at our home anyway, and we never…
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What Does Hemophilia Look Like?
What does an illness look like? Sometimes a bald head, pale skin, or a mobility device are outward signs that someone has an illness. People would never know my sons have hemophilia by looking at them today. My oldest son, Julian, is 24. He lives on his own,…
Hemophilia News Today brought you daily coverage in 2020 of important discoveries, treatment advancements, clinical trial findings, and other important events related to hemophilia. As we look forward to bringing you more news this year, we present here the 10 most-read stories of 2020, along with a summary of…
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on uniQure’s gene therapy program for hemophilia B, which includes AMT-061 (etranacogene dezaparvovec), due to a possibly related serious adverse event in one patient. The event concerns a preliminary diagnosis of hepatocellular carcinoma (HCC), a form…
Funding has been set for the RESET-HA Phase 3 clinical trial evaluating Tremeau Pharmaceuticals‘ TRM-201 (rofecoxib) in people with hemophilic arthropathy — a painful and degenerative disease caused by recurrent bleeding in the joints. The private investment firm Gurnet Point Capital also will support other aspects of TRM-201‘s…
A single dose of the experimental gene therapy FLT180a can safely maintain the activity levels of factor IX (FIX) within a normal range for nearly three years, effectively preventing bleeds and the need for replacement therapies in patients with hemophilia B, according to updated data from a Phase 1/2…