HNF announces Pediatric CMT Natural History Study Enrollment Opening at Two Centers of Excellence – World News Report – Medical Marijuana Program Connection

Global Registry for Inherited Neuropathies

The observed prevalence of CMT symptom onset before age of 16 underscores urgent unmet need to treat this progressively debilitating disease upon diagnosis.

NEW YORK, NY , UNITED STATES, August 31, 2023/EINPresswire.com/ — Charcot-Marie-Tooth (CMT) disease affects 1:3300, nearly 3 million people worldwide, and many remain undiagnosed.

The Hereditary Neuropathy Foundation (HNF), an advocacy and research 501c3 non-profit, today announces a Pediatric CMT Natural History Study enhancement to their Charcot-Marie-Tooth (CMT) and Inherited Neuropathies (IN) patient registry, Global Registry for Inherited Neuropathies (GRIN). GRIN is an IRB approved, patient consented registry. This research consortium consists of researchers and clinical experts, including various partnerships globally (CMT advocacy groups, data scientists, genetic experts and industry).

CMT is a group of inherited disorders with 128 genes responsible for all the CMT subtypes, CMT1A being the most common. GRIN acquires, records, and analyzes patient-reported data and associated genetic reports to identify the burden, diagnostic journey and prevalence of disease. CMT impacts the quality of life starting in childhood and is progressively debilitating. Currently, there are no treatments, but there are many potential therapies in the pipeline. There are still gaps in understanding…