Research into how CMT affects patients’ daily lives has historically been limited. To address this gap, the French company Pharnext partnered with CMT scientific experts and international patient advocacy organizations (PAOs) to study the real-world impact of CMT. The result is the CMT&Me study, a two-year, six-country initiative sponsored by Pharnext.

The study aims to collect real-time data directly from patients describing what it is like to live with CMT. It also seeks to understand how treatment can improve quality of life and slow disease progression.

How the Study Collects Data

Patients participate using the CMT&Me digital app, which uses “bring your own device” (BYOD) technology. Participants use their own smartphones to complete questionnaires and surveys at their convenience, and the app submits responses directly to a central database.

A Scientific Advisory Board oversees the study, including clinicians who care for CMT patients, PAO representatives, and specialists in patient-reported outcomes (PRO) and data management. All patient data is anonymized; researchers who access the database cannot identify individual participants.