My husband and I have always found ourselves living in between places. The first apartment we moved to was in a hub between three cities. Now we live on the border between two cities. Just a few streets away from us is a sign that says, “Thank…

Last year, I met Kayla Mack through a Facebook group for people with von Willebrand disease (VWD). Kayla has type 3, a severe form of the condition. She reached out to the group for help after a terrifying visit to the emergency room. She wrote, “I’m getting very afraid…

Three years after receiving the one-time gene therapy Roctavian (valoctocogene roxaparvovec) at trial, the majority of men with severe hemophilia A in the Phase 3 GENEr8-1 study were off standard replacement therapies and did not experience any bleeds requiring treatment. That’s according to new data announced in a…

I recently read a story about a family who drove to Nevada from Florida, hoping to get home for Christmas. Their flights had been canceled several times, so instead of spending the holiday at home with family, they spent several days on the road. Unexpected airline issues thwarted their plans.

Our New Year’s didn’t go as planned this year. My husband, Jared, our daughter, Cittie, and I were supposed to book a room for a staycation and then go out to the city center to see midnight fireworks. However, that didn’t pan out because the room we had been looking…

One-time treatment with the investigational gene therapy SPK-8011 led to sustained low bleed rates for people with hemophilia A in a Phase 1/2 clinical trial. That’s according to data of up to 5 years of follow-up presented by the therapy’s developer, Spark Therapeutics, at the annual meeting of…

This time of year, I hear many stories of people committing to change and promising to embrace a new way of living. Unfortunately, many of us quickly forget our resolutions. A sense of failure can seep into our lives as, once again, we fail to honor promises made to ourselves.

A cursor blinking on a blank page. A fresh blanket of snow. A blank canvas with new paint ready to be used. Like these images, the new year represents a blank slate and hope for what’s to come. Many people make New Year’s resolutions, such as losing weight, exercising more,…

Preventive treatment with Hemlibra (emicizumab) appears to be safe and helps keep bleeding under control in babies who have severe hemophilia A. The number of bleeds was “zeroed out” in nearly half of the babies, and most had zero bleeds requiring treatment. That’s according to interim results from…

A committee of the European Medicines Agency (EMA) has issued a positive opinion recommending the one-time gene therapy etranacogene dezaparvovec be granted conditional marketing authorization to treat appropriate adults with hemophilia B. The recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) will now be…