Clinical Trials
Clinical trials are a critical step in transforming scientific discovery into effective therapies for Charcot-Marie-Tooth disease.
Through carefully designed and ethically governed studies, clinical trials evaluate the safety, efficacy, and long-term impact of potential treatments. Participation in trials offers patients access to emerging therapies while contributing to the collective understanding of CMT.
ECRA supports awareness, transparency, and collaboration in clinical research. By fostering connections between researchers, clinicians, and patient communities, we aim to facilitate responsible trial development and informed participation.
- Overview (regularly updated): https://clinicaltrials.gov/search?cond=Charcot%20Marie%20Tooth%20Disease
- Report to EUCMTSC 2025: Davide Pareyson, Outcome measurement for assessing iNMD therapies and the effects of new drugs in clinical trials
- Report to EUCMTSC 2025: Tim Vangansewinkel: Running and Planned Clinical Trials