Research Centre for Paediatric Neuromuscular Diseases
As part of its AFM 2017 strategic plan, the AFM-Telethon wished to create a clinical research and management centre for children to facilitate the implementation of clinical trials in young patients with neuromuscular diseases.
Located at the Trousseau Hospital in Paris, I-Motion includes a neuromuscular consultation and clinical research laboratory that leads therapeutic trials and natural history studies.
Leaders
- Odile Boespflug-Tanguy
- Raphaël Vialle
Objectives
- To conduct clinical trials with an international impact and sponsored by industrial, academic or internal partners
- To conduct translational programs in the neuromuscular field (AIM, industrial and academic partners)
- To develop non-invasive tools for diagnosis and evaluation
- To contribute to patient registries and biobanks
- To develop innovative educational approaches at national and international levels
Types of studies conducted at I-Motion
- Studies concerning innovative therapeutic approaches (GT, CT, gene surgery); for CT: the haematology department has the required permissions
- Pharmacological studies
- Development of clinical evaluation criteria and identification of biomarkers
- Observational and natural history studies
- Interventional studies, including rehabilitation
- Registries and databases
