Survey to Improve Clinical Endpoints and Functional Outcome Measures for the Pediatric CMT Patient
Recent advances for possible treatments for Charcot-Marie-Tooth (CMT) disease have set the stage for developing additional clinical trials. As we approach launching clinical trials for the pediatric CMT patient community, we seek to collect data from the thought leaders in the field to help us in our efforts to better understand the patient’s experience of living with CMT, as well as the key issues and challenges facing the professionals working with these patients such as diagnosis, disease characteristics, common symptoms, current treatment options, and risk-benefit balance for the pediatric patient and their families--especially from the perspective of the pediatric neurologist and HCP.
Your input can help us to determine pediatric diagnostic guidelines as well as future clinical trial design with ideal outcome measures and endpoints. Additionally, we would like to collect data determining quality of life trends with children and adolescents such as their ability to stand for long periods of time, climb stairs, walk distances, running, levels of pain and other daily Activities of Living (ADL’s). Your perspective on the impact CMT has on a young patient’s life is vital to moving these trials forward!
Your name will be anonymous and not shared with the public. We hope we can count on your participation in this short survey, and thank you in advance for generously sharing your time and insights with us.