The holiday season brings happiness and joy to many. Homes are adorned with lights and decorations, and people wear clothing in festive colors. The excitement is contagious. However, many people find this time of year to be exhausting and even painful. When they are living with a rare or chronic…

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

My 10-month-old baby, Cittie, is becoming a daddy’s girl. She looks for her daddy when she wants to play. She clings to him when she wants to be carried. She gets uneasy whenever he leaves the room. Jared, my husband, can’t even stand up to go to the bathroom, lest…

Enzyre is teaming up with Takeda to develop a diagnostic device that will allow people with hemophilia to determine their blood coagulation status at home. Under the collaboration agreement, the Dutch diagnostics company Enzyre will receive funding from Takeda to optimize its Enzypad technology. The goal is…

We’re a family that has the good fortune of traveling often. It’s not always a grand journey — most trips are just a few miles from home. One constant during our travels is a set of tiny traveling notebooks — one in my purse and one in my husband’s pocket.

My wife is scheduled for surgery this week in Albuquerque, New Mexico. She will be in the hospital for one or two nights. We live about 3½ hours from the hospital, so we will be gone for about three days. We are optimistic that her recovery will go well…

The holiday season is in full swing, and with fewer shopping days, it will be upon us more quickly than usual. While we are concerned with presents, shopping, parties, and entertaining, it is easy to forget about taking care of ourselves. We can put off our self-care easily by blaming…

A “6 Years Ago Today” moment popped up in my Facebook feed recently. The notification took me back to another Thanksgiving when my son, Caeleb, was 7, and we were in the hospital. He had port access issues, his factor levels were all over the place, and a knee bleed…

My husband, Jared, and I are at a point in our lives where our careers have taken on greater significance. It seems like just yesterday when we were college students eager to graduate and experience life, curious about the mysteries that the future might hold. We were figuring out our…

BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A. The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly…