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…

My husband, Jared, and I run an online jewelry store. Over the years, the fact that our store is run by persons with disabilities (PWDs) has become one of its main selling points. We’ve had fellow PWDs and advocacy supporters speak to us and purchase items. They mentioned that…

Grifols is expanding its commitment to the World Federation of Hemophilia (WFH) Humanitarian Aid Program, donating at least 240 million international units (IU) of blood clotting factors over eight years to consistently treat some 3,000 bleeding disorder patients in developing countries. The contribution, starting in 2022 and running through…

As a caregiver, hope exists in practically my every waking thought. I wish for things to improve regarding my loved ones’ treatment. Hope keeps me going so that I may provide the best care possible for them. Four simple letters put together in the proper order set my heart and…

I have guaranteed quality time with my youngest son, Caeleb, who is 15 and has hemophilia, every Sunday morning. He comes with me to the church where I serve as pastor, and he video records the service each week. Our commute is 40 minutes one way, and in that time…

I recently scheduled a consultation with a psychiatrist after years of trying to manage my mental health without professional help. Since I was a teenager, I’ve dealt with chronic mental health issues that manifested in bothersome ways. In the past, I had difficulty sleeping, disordered eating, and fluctuating moods. I…

This time of year reminds me of hope and the promise of new beginnings. In the spring of 2014, when my son “MacDonald the Younger” was 8, his life drastically changed. Along with our talented hematologist, Dr. Winter, we mapped a treatment plan that proved successful for my young, stinky…

Though the school year is nearly over for my youngest son, Caeleb, who is 15, he just returned to in-person learning for the first time since the pandemic began. Caeleb, who has hemophilia, is a high school freshman and a new student to the school district this…