When I was a young girl, I sat next to my father while he self-infused. I handed him his supplies — butterfly needles, syringes, alcohol, cotton, and medical tape — as he needed them. I watched every step. The factor was mixed, then drawn into…

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 safely and effectively prevented bleeding episodes and the use of clotting factor VIII (FVIII) for up to four years in men with hemophilia A, according to the latest data from a Phase 1/2 trial and its extension study.

AMT-061 (etranacogene dezaparvovec), an investigational gene therapy uniQure is developing to treat hemophilia B, successfully increased factor IX (FIX) activity and controlled bleeding — while markedly reducing the need for other treatments — in the Phase 3 HOPE-B clinical trial, top-line data show. These findings will be presented at…

Higher replacement factor doses and the use of preventive treatments are just two ways prescription practices for hemophilia have changed over time, according to a survey of specialists over 22 years. The surveys also show the impact of Hemlibra’s (emicizumab-KXWH) approval and showcase the expectations for gene therapy,…

I taught choral music in a few local school districts in the Houston metropolitan area in a former life. One year, a friend of mine in the bleeding disorders community asked if I had a group that could provide entertainment at the hemophilia Christmas party. I told her…

Most people probably have a handful of good stories to tell at parties, but someone with a rare medical condition never is at a loss for a new tale to tell. I recently led a workshop to help people in the bleeding disorders community craft their “elevator pitches,” which…

I’m a pastor in the United Methodist Church, where the one constant we share is change. In our denomination, pastors serve on an itinerant system, which means that the cabinet (the bishop plus the district superintendents) chooses which pastors will serve in different locations. As a result, I’ll…

It’s easy to distance yourself from the issues of a bleeding disorder when everything is going well. My youngest son, Caeleb, has not been in the hospital for an overnight stay in several years, and the pain he would endure from repeated bleeds is a distant memory. He…

Biogen recently hired two more executive management team members for Bioverativ, a Biogen spin-off company that is expected to be up and running in early 2017. Bioverativ will focus on the discovery, research, development, and commercialization of new treatments for hemophilia and other blood disorders. Rogerio Vivaldi, MD, is Bioverativ’s new executive…

In Austria, most patients with hemophilia have been vaccinated against COVID-19 — but some expressed concerns over the vaccine’s potential side effects — a small study found. Data showed that 75.0% of the patients in the study were fully vaccinated and that 87.5% had received at least one dose…