CSL Behring has closed an agreement giving it global commercialization and licensing rights to AMT-061, an experimental gene therapy for hemophilia B now being tested in a Phase 3 clinical trial. The therapy’s developer, uniQure, will receive an upfront cash payment of $450 million from CSL…

When I was young, I learned how to be a caregiver for my grandfather. My mother, sister, and I lived with my grandparents for longer than I can remember. I had an incredibly unique relationship with my mother’s parents, particularly my grandfather. When I was about 13, he had a…

Doctors, nurses, hospitals, and clinics are simply part of life with a rare, chronic condition such as hemophilia. Many people in the bleeding disorders community learn early on that speaking up for themselves and their loved ones is crucial to their care. Over the years of raising two sons with…

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…

My husband, Jared, and I are quiet, introverted people. We’re both averse to noisy crowds and overstimulating activities. Our idea of a good time is immersing ourselves in hobbies or getting away to a weekend nature retreat. Although my Myers-Briggs Type Indicator is ENFP, a personality classification that stands…

The last time my youngest son spent the night in a hospital due to a breakthrough bleeding episode was in February 2014. As years passed, his health continued to improve, and life returned to normal. The only difference between my son and the boy next door was the…

Before cellphones, memorizing phone numbers was a necessity. Racing home to make a phone call on the landline was how I made plans with friends and scheduled appointments. With smartphones, I can make a call with the touch of a button, no matter where I am. Although I no longer…

An investigational gene therapy for hemophilia B, called CB 2679d-GT, increased factor IX (FIX) levels and significantly reduced bleeding in a mouse model of the disease, a preclinical study demonstrated. The clotting activity of this potential therapy, being developed by…

In two months, my oldest son turns 25. After the initial shock that he is reaching a milestone birthday, I realized that next year proves crucial in terms of insurance. I currently cover him with my benefits package, but starting at 26, he must provide coverage for himself. He…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…