Plenary sessions and keynote speakers




Marina Cavazzana-Calvo, MD, PhD


Marina Cavazzana-Calvo is a paediatrician, Professor of Haematology since 2000, and the Director of the Department of Biotherapy at Necker Hospital, Paris Descartes University. She also leads a research laboratory at Imagine Institute. Her main research and clinical interests are the development of the immune system, genetic diseases of the hematopoietic system, and cell and gene therapy. She has initiated several clinical trials based on the use of ex vivo gene modified cells to treat patients with inherited disorders. She was awarded the title of Officier de l'Ordre National de la Légion d'honneur in 2011 and received the Irène Joliot Curie “Scientific Women of the Year” award in 2012.




Amy Shapiro, MD


Amy Shapiro, MD, is Medical Director of the Indiana Hemophilia and Thrombosis Center in Indianapolis, and Adjunct Professor of Pediatrics at Michigan State University. Shapiro completed her pediatric internship, residency, and fellowship in pediatric hematology/oncology at the University of Colorado Health Sciences Center in Denver, United States. Author or co-author of more than 300 journal articles, abstracts, and textbook chapters, Shapiro is focused on improving treatment for people with rare bleeding disorders. She has served on the National Hemophilia Foundation’s (NHF) Medical and Scientific Advisory Council. As one of the founders of the American Thrombosis and Hemostasis Network (ATHN), she has served as Co-Chairman of the Board of Directors and remains active on various ATHN committees.



Karen Facey, PhD


Summary: Patients and their informal caregivers have unique knowledge about living with their disease, using health services, and taking treatments. They can share their preferences for future treatment paradigms and tell us what matters to them as we seek to develop patient-centered care. All these experiences, preferences, and perspectives can be used to inform policy and decision making. However, we need to consider the pros and cons of the “patient voice” and reflect on issues of equity and legitimacy to ensure that we involve patients in policy making in a manner that is efficient and effective. We need to reduce the burden on patients when engaging in policy processes and ensure that patient-engagement focuses on the areas that will really make a difference. This presentation will reflect on these issues in relation to the design of healthcare services, research into new treatments, and access to new medicines for hemophilia.



Karen Facey has worked as a senior statistician in pharmaceutical and medicines regulation. In 2000, she set up the first national health technology assessment (HTA) Agency in Scotland. Since 2003, she has been an independent consultant on HTA and patient involvement. She served for eight years as a Non-Executive on the Scottish Health Board and led work on resource allocation for NHS Scotland. She is now a part-time lecturer at the University of Edinburgh. Her research interests include the treatment of rare diseases. In 2005, she founded the HTA International Interest Group for Patient/Citizen Involvement in HTA and is lead editor on the definitive book on patient involvement in HTA.



Claire McClintock, MD


Summary: Mother, sister, daughter, grandma: we bleed too. Exploring the physical and psychological impact of bleeding disorders in women.


Claire McLintock, MD is a hematologist and obstetric physician with interest in thrombosis, haematosis, preeclampsia, recurrent pregnancy loss and medical disorders in pregnancy. Dr McLintock is a Senior Lecturer and Obstetric Physician for the Department of Obstetrics and Gynaecology, University of Auckland Medical School and has given lectures at more than 30 medical conferences. She is a member of council for the Australasian Society of Thrombosis and Haemostasis and President of the Society of Obstetric Medicine of Australia and New Zealand. Dr McLintock is a member of the International Society of Thrombosis and Haemostasis and is co-chair of its scientific subcommittee on women's health issues. She serves on of several international expert panels and advisory boards and has her research published in many medical journals.



Thierry van den Driessche, MD, PhD


Summary: Momentum in the field of gene therapy is increasing, with several gene therapy products being approved by regulatory authorities. Hemophilia has been considered to be a promising avenue for gene therapy since the 1990s. Preclinical studies have demonstrated the efficacy and safety of gene therapy in hemophilic mice and dog models. It is encouraging that sustained therapeutic factor VIII and IX expression levels can now be attained after gene therapy in patients with severe hemophilia A or B—paving the way for a bona fide cure. Nevertheless, there are still challenges that need to be overcome to maximize the benefit of gene therapy for patients with hemophilia.


Professor van den Driessche, MD, obtained his PhD at the Free University of Brussels (Belgium) in 1992 and was a visiting fellow at the Weizmann Institute for Science (Israel) in the field of gene therapy for cancer. He continued his research as a post-doctoral fellow at the National Institutes of Health (U.S.A.) in the lab of gene therapy pioneers: R. Michael Blaese, MD, and Rick Morgan, MD. In 2010, he became Professor at the University of Leuven and Full Professor at the Free University of Brussels, where he is now Director of the Department of Gene Therapy & Regenerative Medicine. van den Driessche has also served as President of the European Society of Gene & Cell Therapy. His main research focuses on gene therapy for hereditary diseases—particularly hemophilia—and the development of improved gene therapy and gene editing technologies. Through preclinical models, his team has postulated that hemophilia A can be cured by gene therapy.


The WFH 2018 World Congress is supported by the Glasgow City
Council and VisitScotland

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