SPK-9001, an investigational gene therapy being developed by Spark Therapeutics and Pfizer, continues to demonstrate great potential to treat hemophilia B patients in an ongoing Phase 1/2 clinical trial. The latest results showed that the annualized bleeding rates of all treated patients were reduced by 98%. In…

Diabetes and infection with human immunodeficiency virus (HIV) increase the risk of high blood pressure (hypertension) among men with hemophilia, a U.S. study found. Given that both hypertension and diabetes can lead to a brain bleed in hemophilia patients, these findings support early screening for these conditions in this…

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…

Due to the global COVID-19 pandemic, World Hemophilia Day (WHD) will be  a little different this year to keep everyone safe. Still, there are multiple ways to participate in the April 17 event intended to increase awareness and understanding of hemophilia and other bleeding disorders. Begun in 1989 and…

Home improvement is a popular hobby here in the Philippines. Over the past few years, online communities dedicated to home decorating and renovation have sprouted up all over social media. Lockdown boredom inspired people to find new hobbies and pastimes, and as work shifted from office to home,…

In contrast to observations in the general population, blood type and levels of von Willebrand factor (VWF) don’t appear to influence factor VIII (FVIII) levels in people with non-severe hemophilia A, a study reports. Moreover, age was found to play only a minor role in FVIII levels in…

Sigilon Therapeutics and the Massachusetts Institute of Technology will work together to develop programmable cell therapeutics for the treatment of hemophilia, lysosomal storage diseases, and other serious chronic illnesses. The collaboration will combine Sigilon’s proprietary cell technology with synthetic biology approaches developed by Ron Weiss, PhD, a…

I arrived late for the concert. An usher whisked me to my seat, as my husband and our son Caeleb had already arrived. As I walked down the aisle, feeling very conspicuous in my clerical collar (I didn’t have time to change), I noticed the backs of hundreds…

The U.S. Patent and Trademark Office has granted uniQure a third patent covering the formulation and use of the company’s factor IX Padua gene therapy for the treatment of hemophilia and other bleeding disorders. The new patent (10,465,180) will broaden uniQure’s exclusivity rights over the FIX-Padua encoding gene,…

A Phase 3 clinical trial of treatment candidate AMT-061 for severe and moderately severe hemophilia B has treated its first patient. The open-label HOPE-B study (NCT03569891) is testing the safety and effectiveness of AMT-061 in adult men with hemophilia B. Patient recruitment is continuing for an estimated total of 56…