Hemophilia

Walgreens’ Ray Tancredi Weighs in on Expensive Hemophilia Gene Therapies in the Pipeline

May 13th 2022, 5:59pm

Video

Gene therapies in the pipeline to treat hemophilia will be very expensive, but they also seem to be very efficacious, and multiple could hit the market between now and 2024, said Ray Tancredi, RPh, MBA, CSP, divisional vice president, specialty pharmacy development and brand Rx/vaccine purchasing, Walgreens.

Hemophilia A Gene Therapy Reduced Bleeding Events, Need for Clotting Factors

March 16th 2022, 9:00pm

Article

The phase 3 trial of valoctocogene roxaparvovec for severe hemophilia A showed it reduced bleeding events as well as the use of factor VIII concentrates.

Gene Therapy for Hemophilia B Shows Positive Results in Phase 3 Trial

June 25th 2021, 1:30pm

Article

The company, uniQure, said it is the largest group of patients with hemophilia B to receive a single gene therapy investigational product so far. 

Study Finds Inhibitors Develop in Nonsevere Hemophilia A Earlier Than Previously Reported

October 30th 2020, 8:09pm

Article

Up until now, it was thought most patients with hemophilia A developed inhibitors in middle-age. A new study suggests it usually happens in the early teens.

Gaps Remain Between European Regions for Hemophilia Care Despite Improvements

July 17th 2020, 10:00pm

Article

Hemophilia care principles have been on the rise in Europe over the course of the last decade, however, differing regions and policies on the continent reveal gaps in access to various treatments and specialty services.

Prevalence of Hemophilia Worldwide Is Triple That of Previous Estimates, New Study Says

September 10th 2019, 1:00am

Article

The meta-analysis showed that more than 1,125,000 men around the world have the inherited bleeding disorder; 418,000 of those have a severe version of the mostly undiagnosed disease. Previously, only 400,000 people globally were estimated to have hemophilia.