BioMarin Pharmaceutical  will add an additional Phase 3 study to the clinical development of its investigational gene therapy BMN 270 for hemophilia A. BioMarin will conduct two separate trials testing the effectiveness and safety of two different doses of BMN 270. Both trials are expected to initiate in the…

Xenetic Biociences has received a $3 million milestone payment from its partner Shire for advancing the Phase 1/2 clinical trial of PSA-recombinant SHP656, a factor VIII protein, evaluating the drug’s effectiveness as a long-acting therapy for hemophilia A. Shire’s main objective behind the agreement is to develop a novel FVIII…

Express Scripts has teamed up with PinnacleCare to include in its new Rare Conditions Care Value (RCCV) program a service that provides patients with rare diseases like hemophilia a second opinion from a specialist care provider. The initiative, simply called Second Opinion, is designed to give patients an additional…

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,…