Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

For many years we tried to overcome my younger son’s reaction to factor VIII. No matter which medical product we tried, we ended up with the same results. He would break out in hives early in the infusion. We learned that my son had developed an allergy to factor VIII,…

Over the past few months, my son Caeleb has complained of knee pain. “It’s not buzzy or achy, Mom,” he would say. “It just really hurts.” After his annual visit to a hemophilia treatment center this summer, his doctor recommended an MRI of Caeleb’s knee and ankle. Because…

I will never forget the evening in the mid-80s when my parents sat my brother, my sister, and me down for a critical conversation. They told us that my father had HIV. There was a very good chance he would die before my sister graduated from…

I am a work-from-home mom, which means that I have two full-time positions as a “mom” and a “worker.” I am also a full-time carer to my husband with hemophilia. Thankfully, that position affords me several “leave credits” as my husband is not always injured. (Hopefully, the investment we’ve made…

Marstacimab, an experimental treatment in clinical testing for hemophilia A and B, was able to restore clotting in hemophilic blood and plasma tested in laboratory assays, according to a study. If proven safe and effective in patients, the investigational therapy could provide an alternative preventive solution to…

I was with my son in a store recently when a coloring book for adults caught my eye. Ordinarily, I wouldn’t have given it a second glance. But what made this one special was the title on its cover: “The Road Not Taken.” I couldn’t resist opening the book.

It’s easy to look back and think about what one could have done differently. Handling a crisis with grace rather than anger, calling the doctor immediately instead of waiting, or keeping a diagnosis secret to avoid others’ judgment. We all make mistakes and, hopefully, we learn from them. Forgetting…

Many of us have been there. We’ve walked into a medical office in pain, seeking relief. As the doctor completes an evaluation, we are asked that common and potentially problematic question: “Please rate your pain on a scale of 1 to 10.” I hate pain…

It’s quite difficult to focus on any sense of positivity when bleeds become more frequent. After a bleed-free streak that lasted over six months, it was easy to be disappointed when hemophilia made me feel vulnerable and weak again. It’s not all that bad, though. At times, being bedridden results…