A single administration of AMT-061, an investigational gene therapy for people with severe and moderately severe hemophilia B, increased therapeutic levels of factor IX (FIX) in all patients enrolled in uniQure’s ongoing Phase 2b trial, the company announced. Ultimately, this trial’s purpose is to confirm the dose of AMT-061…
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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…
The first patient has been dosed in China in a registrational clinical trial of BBM-H803, a one-time investigational gene therapy that Belief BioMed is developing to prevent bleeding in hemophilia A. The Phase 1/2/3 clinical trial (CTR20233400) is testing how safe and well tolerated BBM-H803 is as a…
Researchers from the PharmedOut project at Georgetown University Medical Center analyzed how pharmaceuticals market directly to people with hemophilia and found that, while strategies are effective, further regulation and studies are necessary. The study, “Direct-to-consumer Marketing to People with Hemophilia,” was published in the PLOS Medicine Policy Forum. PharmedOut involves doctors,…
My biggest fear for my sons, who live with severe hemophilia, is an auto accident. When my oldest son, Julian, began to drive, I impressed upon him anew the importance of wearing a MedicAlert emblem. He wore it like dog tags for years when he was younger,…
Growing up in suburban Milwaukee, I saw the world through a self-employed lens. My grandfather sold and repaired typewriters, a business he started in downtown Chicago in 1951, a time that must have been most colorful. My family settled in Milwaukee, where my father became the largest independent distributor of…
A few weeks ago, my oldest son, Julian, called me from the Blue Ridge Mountains in North Carolina and informed me that he had throbbing pain in one of his teeth. He immediately went to a nearby dentist and got news he didn’t want to hear: The tooth…
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Roger Ailes’ Journey With Hemophilia
On May 18, Roger Ailes, the former CEO of Fox News passed away at his home in Florida after suffering a fall. The 77-year-old was diagnosed with hemophilia at the age of two, after he fell, bit his tongue and couldn’t stop bleeding. MORE: Six self-care tips for people living with…
I recently came across a social media post that hit home. It began: “Never judge someone with a chronic illness if they …” and went on to list things like skipping meds, eating foods that might trigger symptoms, resting all day, or even doing something that looks “wrong”…
Dear Santa, This year, I’ve decided to pen a special letter to you — not for myself, but for the global hemophilia community. As the holiday season fills the air with hope and goodwill, I can’t help but reflect on the challenges and dreams of those of us living…