My favorite shows are “Criminal Minds,” “Bones,” and the “Law and Order” franchise. In these police procedural crime dramas, the suspense and action are exciting and the plot twists surprising. Episodes regularly depict hostage situations. Watching actors portray the fear and horror one must…

Results of the first group of patients with hemophilia B treated with Catalyst Biosciences’ lead candidate CB 2679d in a Phase 1/2 clinical trial demonstrated its improved stability and activity compared to Pfizer’s BeneFix standard-of-care replacement therapy. CB 2679d (also known as ISU304), was designed to be the…

Watching gymnasts Nadia Comaneci and Olga Korbut in the 1976 Olympic Games inspired me to do front rolls in my backyard. I knew back flips and aerial twirls weren’t in my reach, so I set my sights on mastering the cartwheel. But it was never to…

One fall evening in 1998, I looked around my dining room table in Houston and saw the faces of men and women who’d become some of my dearest friends over the past year. Laughter flooded the room as we stood in awe at what we’d accomplished. I stood, raised a…

If there’s one thing that moves me to tears, it’s sharing the love I have for my sons. I think of the million and one directions that once lay before me and how I chose to follow my heart, marry the love and joy of my life, and raise children.

Although moderate to severe hemophilia patients are generally protected against the development of cardiovascular disease, cardiovascular-related events can still occur in this patient population, a study reports. These findings suggest that efforts should be made to prevent cardiovascular disease in hemophilia patients who have risk factors associated with the…

Some years back, my two sons packed their suitcases and Caeleb, the youngest, said, “All right, it’s time to go on a Daddy-and-son madcap adventure.” My wife had a speaking engagement out of town, which meant the MacDonald boys were going to set out on their own, together on the…

Specific genetic changes may account for a larger proportion than previously thought of children with severe hemophilia B who develop inhibitors against coagulation factor IX replacement therapy, according to an international study. The study, “Inhibitor Incidence In An Unselected Cohort Of Previously Untreated Patients With…

Tremeau Pharmaceuticals has received guidance from the U.S. Food and Drug Administration (FDA) on how to conduct a pivotal Phase 3 trial exploring rofecoxib (TRM-201) in patients with hemophilia-associated joint pain. The advice was offered during a formal meeting with the agency, where both parties agreed on the design…

Novo Nordisk expects a decision this month by the U.S. Food and Drug Administration (FDA) on its product N9-GP  (nonacog beta pegol) as a treatment for hemophilia B. Approval of the drug may depend on the conclusions the FDA makes regarding safety concerns raised by its blood products advisory committee,…