AMT-061 (etranacogene dezaparvovec), uniQure‘s investigational gene therapy for hemophilia B, is safe and effective — even in patients with pre-existing antibodies against the treatment’s viral carrier, according to new trial data. Data from the Phase 3 HOPE-B trial suggest that nearly all hemophilia B patients, regardless of pre-existing viral…

People are required to earn a certification or license for numerous activities. To drive a vehicle, a person must secure a learner’s permit or driver’s license. To become a lifeguard, a person must go through training to earn a certification. Even working a part-time job in the food industry…

“Beep, beep.” I leap out of bed at 10 a.m. on a Saturday. My plans for the day include geocaching, a wonderfully nerdy hobby in which I hike through forests and urban areas to find hidden boxes. The GPS on my phone leads me…

Screening for clinical trials in gene therapy can exclude the vast majority — 92% — of severe hemophilia patients, for reasons too often due to potentially modifiable factors such as socioeconomic status and location, a study of patients at a treatment center in Belgium reported. Patients’ unwillingness to…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on uniQure’s gene therapy program for hemophilia B, which includes AMT-061 (etranacogene dezaparvovec), the company announced. According to uniQure, the FDA found it was “very unlikely” that AMT-061 had contributed to a case of liver cancer in a patient who…

My husband, Jared, has a complex medical situation: He has severe hemophilia B and a seizure disorder, which both greatly affect his daily life. He recently started online consultations with a psychologist, a psychiatrist, and a neurologist to help him work through his seizure disorder, which was caused by a…

Healthcare costs can be 25 times higher for hemophilia B patients than for individuals who do not have a bleeding disorder, according to a recent study. These findings reflect both direct costs of hemophilia treatment and a greater need for other healthcare resources, researchers noted. The study, “…

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…

The paladin By-Tor from the Order of Light fights to protect others and always chooses life over death, even when it places him in certain danger. In the world of Dungeons and Dragons, he uses his sword and…