Gene therapy delivering the blood clotting factor VIII (FVIII) — whose lack causes hemophilia A — into the joints did better at protecting against hemophilic arthropathy (joint damage) than did administration into the bloodstream, a study in mice suggests. Its findings support the potential use of FVIII injected directly into…

In this video shared by Colleen, learn about a mobile app from Pfizer that helps hemophilia patients track bleeds, infusions and factor supply. It also allows those living with the disease to track their daily physical activities, steps and heart rate to help…

A single dose of Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and the need for prophylaxis, or preventive therapy, for as long as seven years — the longest follow-up to date for any hemophilia A gene therapy clinical trial. While most of the 12 patients…

US Bioservices, a specialty pharmacy, announced that it is working with the pharmacy benefit management company MedImpact to dispense prescriptions and select services through the MedImpact Direct Specialty Program for the hemophilia community. Under the collaboration, US Bioservices will offer hemophilia patients who are MedImpact members the opportunity to…

Last Sunday, my oldest son came to town for a photoshoot. He hopes to build a strong website presence so that directors and casting agents consider him for future projects in musical theater. We made plans to eat lunch before meeting with the photographer. My youngest son joined me, and…

Mobility problems, bleeding, pain, and uncertainly in daily life are the biggest challenges people with hemophilia are facing, according to a survey from six central European countries. Additionally, while most respondents, including caregivers, felt well informed on hemophilia, they continue to seek further education on the condition and new…

People with hemophilia are more likely to be infected with the SEN virus (SENV), which is associated with blood transfusion hepatitis, or liver inflammation, than healthy people, a study in Iran suggests. The virus was detected in more than half of patients with hemophilia A and most commonly in…

An international team has joined efforts to establish guidelines for effectiveness and outcome measurements regarding gene therapies in hemophilia. The CoreHEM project will be led by researchers from McMaster University in Ontario, Canada, in collaboration with the National Hemophilia Foundation (NHF) in the U.S. and the Green Park…

A research team from the departments of biochemistry and pathology at the University of Pennsylvania’s School of Dental Medicine has found a successful technique using genetic engineering to coax lettuce and tobacco plants to produce foreign proteins in their leaves. The applications include producing the human clotting factor to make…

One of my favorite movies is “Galaxy Quest,” a comedy that parodies and pays homage to science fiction films and TV. In it, the cast members of a cult series much like “Star Trek” are drawn into an interstellar conflict by real aliens who think the series is…