Clinical Research

In 2020, more than 30 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.