Clinical trials

The Clinical trials pole is the core of the activity of the Institute of Myology. It includes two clinical trial platforms for neuromuscular diseases, I-Motion Pediatric and I-Motion Adults, and the Registers and Databases pole, dedicated to the development and follow up of databases, registers, observatories and cohort monitoring. The clinical trial platforms allow the clinical management of patients, children or adults, the evaluation of new treatments and the performance of innovative clinical trials.

Currently, more than 50 research protocols are being developed at the Institute of Myology: clinical studies (natural histories, management, rehabilitation training) but also therapeutic trials (pharmacology, cell therapy, gene therapy, enzyme therapy).

In addition to trials driven by the teams of the Biotherapies Institute for Rare Diseases, created by the AFM-Telethon, the scientific and technological platform of the Institute is recognised internationally by pharmaceutical companies or organisations (US NIH, Genzyme PTC Therapeutics, Santhera, Roche…), which have chosen it as the principal investigator centre for some of their trials.