Gene Therapy Researcher Honored by Children’s Hospital of Philadelphia
Katherine High, MD, has received the gold medal from Children’s Hospital of Philadelphia (CHOP) in recognition of her work to…
Articles by Marisa Wexler, MS
Roche Extends Partnership With WFH Aid Program
Roche has extended its partnership with the World Federation of Hemophilia (WFH) to expand access to treatments for…
Europe Awaits Proposed New Framework for Sharing Health Data
The European Commission is expected to propose a new governing framework for health data next month, called the European Health…
Female Carriers Show High Rates of Bone Disorders in Large Study
Osteoporosis and other bone disorders are more common in hemophilia carriers than in the general population, according to an analysis…
Roctavian, Gene Therapy for Severe Hem A, Safely Raises FVIII Levels
Treatment with the experimental gene therapy Roctavian (valoctocogene roxaparvovec) significantly increased factor VIII levels in men with severe…
Dose-confirmation Study Launched for Hem B Gene Therapy FLT180a
The first participant has been dosed in a Phase 1/2 dose-confirmation trial that is testing FLT180a, an experimental gene therapy…
Hemophilia Carriers Report Heavy Periods, Little Empathy From Doctors
The vast majority of women who have a bleeding disorder or are hemophilia carriers experience noticeable symptoms, such as…
Hemophilia Patients May Face Lower Risk of Heart Disease: 5-year Study
People with hemophilia may be less likely to develop cardiovascular disease (CVD) — disorders of the heart and blood…
Gene Therapy EtranaDez Allows Hem B Patients to Stop Prophylaxis
Nearly all men with moderate-to-severe hemophilia BÂ given the experimental gene therapy EtranaDez (etranacogene dezaparvovec) in the HOPE-B trial have…
Hemophilia A Patients in Roctavian Trial Largely Bleed-free at Two Years
More than 80% of the men with severe hemophilia A treated with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) in…