CMT&Me Study

HNF has been on the forefront of conducting patient-centered research to get a better understanding of how CMT affects the lives of patients. Collecting Patient Reported Outcomes (PROs) is critical in accelerating therapies.

Pharnext, a leading pharma company dedicated to bringing treatments to CMT patients is conducting in partnership with CMT scientific experts and patient advocacy organizations an international study on the impact of CMT on patients’ daily lives. The result of that partnership is the CMT&Me study, which Pharnext is sponsoring over two years in six countries. The aim of the study is to collect real time data directly from patients, who describe what it is like to live with CMT. The study also aims to find out how treatment can improve patient quality of life and slow CMT progression.

The study collects data directly from patients using a digital app, CMT&Me, which collects real-world data using “bring your own device” (BYOD) technology—participants use their own smartphones to complete questionnaires or surveys at their convenience. The app immediately submits the information patients provide to a central database. A Scientific Advisory Board oversees the CMT&Me study, and includes clinicians who care for CMT patients, PAO representatives, and experts in patient-reported outcomes (PRO) and data management. All patient data remains anonymous, and researchers who access the CMT&Me study database will not be able to identify patients.