The Inherited Neuropathy Consortium (INC), which is part of the NIH Rare Diseases Clinical Research Network (RDCRN), has established an international patient contact registry, making it possible for researchers to find new treatments, create new studies and work to improve the lives of everyone with CMT.
Enrollees in the patient registry are contacted when there are opportunities to participate in clinical trials and other research studies such as 6601: Natural History Evaluation of Charcot Marie Tooth Disease.
Enrollment in the patient contact registry is currently being facilitated through INC-affiliated CMTA Centers of Excellence. We strongly encourage anyone who wants to be considered for clinical trials to visit a CMTA Center of Excellence where people with CMT can participate in clinical research being conducted by the Inherited Neuropathies Consortium (INC) with CMTA sponsorship.
The Rare Diseases Clinical Research Network is funded by the National Institutes of Health and the Office for Rare Diseases Research. The INC is a multi-center clinical research collaborative within the RDCRN created to address the following issues pertaining to CMT:
As we near clinical trials, a clinical scale that is sensitive enough to measure successful outcomes needs to be refined;
Patients with different kinds of CMT must be evaluated in a uniform manner so that high-quality clinical data is available to researchers developing clinical trials and studying CMT in humans; and,
Researchers will have to identify genetic modifiers that contribute to variations in patients with identical CMT mutations, even within the same family.
The INC database is critical to complement STAR’s progress and promise for an eventual treatment. In June 2019, NIH announced $5 million in renewed funding for INC.
The CMTA, along with the Muscular Dystrophy Association, CMT United Kingdom, and TREAT NMD (the European Neuromuscular Network), are the partnering patient advocacy groups within the INC. The CMTA is well positioned to facilitate patient recruitment and involvement in cutting-edge research. To that end, we also encourage you to complete a CMTA Patients as Partners in Research Profile so that we can keep you informed about current research and opportunities to participate.