[What is the cross-disciplinary pathway for a patient receiving gene therapy?]

Abstract

The Department of Hematology-Immunology, headed by Professor Jean-Hugues Dalle, is the leading department in France for the care of patients receiving pediatric hematopoietic stem cell transplants. Since 2018, it has been a "major player" in the development of innovative treatments, such as gene therapy. To date, it is the only care service in France, which offers this treatment to stop the neurodegenerative progression of two genetic diseases, in collaboration with the neuropediatric service of the Kremlin-Bicêtre hospital headed by Prof Kumaran Deiva, in conjunction with the national reference center for rare disaeses didicated to leucodystrophies. It is a complex care that requires all the actors a cooperation (parents-children-caregivers).

Keywords: care; circuit transversal; gene therapy; innovative treatment; pediatric; prise en soins; pédiatrie; thérapie génique; traitement innovant; transverse circuit.