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…

As we prepare to celebrate Christmas, I can’t help but remember a particular year in the MacDonald household. In 2013, my youngest son, Caeleb, couldn’t catch a break from hemophilia. Constant spontaneous bleeding episodes into his right knee and ankle kept my boy in the hospital more often…

I am not a fan of roller coasters. Dropping straight down in a car on a wooden track makes me feel like my heart has left my body. Why would anyone want to feel that way? However, I have one exception: the Rock ‘n’ Roller Coaster Starring Aerosmith…

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended Hemlibra’s (emicizumab) approval be expanded to include people with moderate hemophilia A without inhibitors. “We’re very pleased that the CHMP’s recommendation brings us closer to potentially transforming the day-to-day lives of people in the…