I am a fierce advocate for my sons. I question doctors professionally. I say no and ask for other options. I have even found new research and treatment options to offer physicians. I do not give up and even risk being known as one of those moms. If I…

This year, my flower garden has grown exponentially. A wonderful gift that “bloomed” from my COVID-19 boredom was the chance to develop my horticultural skills. I increased my rose collection from six bushes to 14, and I added sunflowers, teddy bear sunflowers, zinnias, wildflowers, poppies,…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

I have been struggling at home lately. I always pictured a peaceful and fulfilling married life. As a wife, I thought I would be queen of my home. Each corner of our house would be filled with my favorite decorations, and our fridge, pantry, and bar would be…

Wouldn’t it have been nice to receive an instruction manual when first diagnosed? The doctor might share a blueprint, surefire way of raising a child with a bleeding disorder: “Follow this when this happens, and all will be fine.” Those who manage chronic illnesses know full well that there…

The terrible, awful, no-good, very bad year for my family is not 2020. It was 2013. My youngest son, Caeleb, spent most of that year in the hospital. In 2013, Caeleb, who was in second grade, missed about 70 days of school due to recurring bleeding in his…

Women with genetic blood disorders, including those who are carriers of hemophilia A and B, face multiple uncertainties surrounding their diagnosis, according to a recent interview-based study. Many women claimed to have been misdiagnosed initially and did not receive a correct diagnosis until after the disease had…

We finally got a dog! She is a 3-month-old purebred golden retriever. It was love at first sight when we saw her at the pet store. From our very first interactions, we knew she was the one. We named her Lucky because the golden color of her coat is associated…

The National Institutes of Health (NIH) awarded the DNA Medicine Institute (DMI) a $1.5 million grant to advance a quick and easy way of monitoring factor VIII (FVIII) and Hemlibra (emicizumab) blood levels in people with hemophilia A, especially those in developing countries. DMI,…

The first participant was dosed with the investigational gene therapy treatment SB-525 (giroctocogene fitelparvovec) in a Phase 3 clinical trial in men with hemophilia A, according to developers Pfizer and Sangamo Therapeutics. AFFINE (NCT04370054) is a pivotal trial, which means it is intended to…