After many years, gene therapy in patients with haemophilia has started to show promising results in alleviating symptoms and improving the disease phenotype.
Medscape has launched a new education program for providers seeking to enhance their knowledge of gene therapy for haemophilia. “Clinical Advances in Gene Therapy for Haemophilia” is presented through a collaboration between Medscape, the European Haemophilia Consortium, the National Hemophilia Foundation and the World Federation of Hemophilia. The program is supported by an independent educational grant from BioMarin.
This collection of programmes provides important information on the science behind gene therapy for haemophilia, the latest safety and efficacy data from clinical trials, and the potential role for gene therapy in addressing the challenges of current treatment methods. Currently, healthcare providers can test their knowledge through two modules on ‘Why Gene Therapy in Haemophilia? Exploring the challenges of current therapies,’ and ‘The Science of Gene Therapy in Haemophilia.’ These two modules are eligible for continuing medical education credits.
Visit the “Clinical Advances in Gene Therapy for Hemophilia” homepage to access the following resources and activities on gene therapy for haemophilia.
If you are not already registered, visit Medscape.org and register free of charge.
Jim is aged 66 and has Haemophilia A with a high titer inhibitor which he developed at age 14.
Maria Elisa Mancuso (MD, PhD) is a Haematologist and works as a Senior Haematology Consultant at the Center for Thrombosis and Haemorrhagic Diseases of IRCCS Humanitas Research Hospital in Rozzano, Milan, Italy. She is Adjunct Clinical Professor at Humanitas University. She obtained a post-degree in Clinical and Experimental Haematology and a PhD in Clinical Methodology. She is involved in clinical research and has published several original articles in peer-reviewed journals a The Lancet, Blood, Journal of Thrombosis and Haemostasis, Haematologica, Thrombosis and Haemostasis, British Journal of Haematology and Haemophilia. She is reviewer for several peer-reviewed journals and member of the Editorial Board of JTH. She is a member of several scientific societies (ISTH, WFH, ASH, EAHAD, SISET, AICE) and was a medical member of the Inhibitor Working Group of the European Hemophilia Consortium. She is co-chair of the ADVANCE Study Group. She has acted also as co-chair of the Scientific and Standardization Subcommittee of ISTH on FVIII, FIX and rare bleeding disorders. She has been involved as principal and co-investigator in several clinical trials, and she takes care of both children and adults with hemophilia and other congenital bleeding disorders with a specific scientific interest in novel therapies, prophylaxis, inhibitors, and chronic hepatitis C.
As a patient with factor II deficiency, the diagnostic and treatment of rare bleeding disorders is a matter dear to my heart. My motivation to participate in the work of the ERIN committee is to improve both diagnostic and treatment for patients with rare bleeding disorders across Europe.
Economist and financial expert by profession, executive coach and trainer by passion and haemophilia advocate by every drop of my blood through my son (who has severe haemophilia A with inhibitors). Bringing a good decade of practical experience from the corporate insurance world, laser focus, growth mindset and resilience from my own experience, offering you anything I can just do, in hope that together we can make life more fulfilled for those impacted by bleeding disorders.
Amy Owen-Wyard is a Registered Mental Health Nurse. With experience working with children, young people, their families and adults with severe and enduring mental health conditions. Amy was also involved in a service improvement to provide a holistic care approach for those in general hospitals to support both their mental and physical health, whilst sharing her expertise and knowledge in mental health with the wider multidisciplinary team.
