uniQure plans to advance its investigational gene therapy AMT-061 into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B. Gene therapies are being develop to treat diseases caused by genetic mutations. It involves replacing the faulty gene in patients with a healthy gene. AMT-061…
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It’s been nearly five months since I became a mom to baby Cittie. In those months, my life has been transformed into something completely different. My social media feed, which used to be full of items about academics and career, is now full of articles about babies and…
People whose hemophilia A is mild or moderate can show marked alterations to their joints on MRI scans, particularly in the ankles, despite having few joint bleeds, a study reported. These findings highlight the need for closer monitoring of joint health and a likely need for preventive treatment approaches in patients…
Fat-shaming is a widespread issue that can also affect people with hemophilia, especially young children. At its core, fat-shaming involves making negative comments or judgments about a person’s weight, which can lead to feelings of inadequacy or embarrassment. While these remarks may be intended to encourage weight loss or…
The Hemophilia Federation of America recently hosted its annual symposium in Indianapolis. Despite my desire to attend, circumstances didn’t align for me this year. One of the event’s highlights was the debut of digital content agency Believe Limited‘s latest cinematic offering, “On the Shoulders of Giants,” now…
On Sunday mornings in my home, I usually watch “CBS Sunday Morning,” hosted by Jane Pauley. Last week, an interview caught my eye. The segment featured Dr. Jennifer Doudna, who won the Nobel Prize in chemistry in 2020 along with fellow researcher Emmanuelle Charpentier for developing…
TiumBio is preparing to launch a Phase 1b clinical trial to test TU7710, its experimental treatment to manage bleeding in people with hemophilia who have inhibitors, that is, neutralizing antibodies that can lower standard therapies’ effectiveness. “Our team has been developing TU7710 as an innovative hemophilia treatment option,”…
US Bioservices, a specialty pharmacy, announced that it is working with the pharmacy benefit management company MedImpact to dispense prescriptions and select services through the MedImpact Direct Specialty Program for the hemophilia community. Under the collaboration, US Bioservices will offer hemophilia patients who are MedImpact members the opportunity to…
My husband, Jared, has had hemophilia since he was born. He has known no other life than one with a bleeding disorder. And that is why he does not consider hemophilia to be his “worst enemy.” Instead, he considers epilepsy his greatest foe.
The world often feels like an amusement park ride that does nothing but turn in circles, and we must gather the courage to get on. At first, everything spins at a moderate pace, but as the ride continues, the pace picks up, and suddenly everything spins so fast we can…