Hereditary Neuropathy Foundation

Research programs are as follows: the therapeutic research in accelerated discovery (triad) program focuses on funding research grants and venture philanthropy (vp) that supports the development of drug discovery, gene therapy approaches that have the most chance of success. Through partnerships with academic universities, contract research organizations, and industry leaders in the healthcare, biotech, and pharmaceutical sector; hnf aims to support translatable therapy development enabling a pre-ind package and for support of clinical trials. The global registry for inherited neuropathies (grin) patient registry collects robust natural history studies for all subtypes of cmt. Grin is the only cmt database that is all inclusive collecting patient-reported and caregiver surveys, curated genetic diagnosis reports and clinical data which is essential to drug discovery and clinical trials. Grin is global, scalable and supports all 125+ cmt-related genes with over 165 associated mutations. The data has proven to be essential in clinical trial readiness, understanding genotype/phenotype and for clinical trial recruitment. The cmt genie provides the opportunity for patients and clinicians to connect with genetic counseling via telehealth and subsequently the option to receive a genetic test kit through the mail. Patients then have the option to schedule a follow-up counseling appointment to discuss the results and upload them into the secure grin portal. The cmt genie provides faster and more equitable access to critical genetic care for the cmt community and their health care providers.