Gene therapy for hemoglobinopathies, the viewpoint of a specialist in pediatric hemoglobin disorders

Speakers: Mariane de Montalembert

Professor Mariane de Montalembert received her MD and her PhD in Ethics from the Paris Descartes Medical School. She specialized in pediatrics, statistics (option clinical research), red cell diseases, especially hemoglobinopathies, in children, and transfusion She got Habilitation to Direct Research.

She is co-responsible for the Hemoglobin Diseases Unit at the Necker University Hospital, which follows 700 children and the «ROFSED» healthcare network for SCD children in the Parisian area (Therapeutic Education Unit for 700 children and families).

She regularly teaches courses for the European School of Hematology.

In 2013, she developed an online course in French covering the care of adults and children with sickle cell disease (70 hours) and offering validated training in therapeutic education (40 hours). Around 300 healthcare professionals from France, Africa and the French West Indies have taken this course and graduated from the University of Paris.

Current affiliation: Department of General Pediatrics and Pediatric Infectious Diseases, Sickle Cell Center, Necker-Enfants Malades Hospital, Assistance Publique - Hôpitaux de Paris (AP-HP), Université Paris Cité, Paris, France, and Laboratory of Excellence GR-Ex, Paris, France.

Prof de Montalembert is a member of the French Society of Pediatrics, in which she has chaired for more than 10 years the Ethic Committee, of the French Society of Hematology, the European Reference Network in rare Haematological Diseases (EuroBloodNet), the European Association (EHA), and the American Society of Hematology (ASH). She has been member of the EHA Board for the period 2020-2024 and she coordinates the EHA Topic in Focus group on hemoglobinopathies since 2023.

She is member of the Cochrane commission since 2014, and was member of Blood Editorial Board 2021-2024