HNF is advancing and supporting clinical research and trials for CMT.
HNF has been instrumental in advancing and supporting clinical trials by providing valuable patient insight, innovative data collection, and regulatory initiatives to assist in industry-sponsored clinical trial design and to inform the FDA for decision-making and oversight during drug development and commercialization of drugs and gene therapies for CMT.
Learn about clinical trials and how you can participate.
Without your participation, researchers won’t have the essential patient information to develop drugs, gene therapies, and clinical trials for Charcot-Marie-Tooth and other Inherited Neuropathies. In addition, as GRIN grows, we gain greater insights from you as patients to help accelerate therapies for Charcot-Marie-Tooth (CMT) and Inherited Neuropathies.
CMT Thrive Webinar:
The Key Role CMT Patients Play in Research
Presented by: Joy Aldrich, HNF GRIN Patient Registry Coordinator
Pediatric CMT Natural History Study enhancement to their Charcot-Marie-Tooth (CMT) and Inherited Neuropathies (IN) patient registry, Global Registry for Inherited Neuropathies (GRIN).
DTx Pharma Receives FDA Orphan Drug Designation for DTx-1252 for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Dtx today announced that the FDA has granted Orphan Drug Designation to DTx-1252, an investigational FALCON small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Pharnext announces new positive results from the ongoing open-label extension study of their drug PXT3003
Applied Therapeutics, Inc. has announced that their drug candidate, AT-007 (Govorestat), has received orphan medicinal product designation
The study highlights the significant impact of neuropathic pain on the quality of life and psychosocial well-being of individuals with CMT.
Mitochondria are the powerhouses of our cells. Think of them as our body’s batteries. Mitochondrial disease causes these batteries to run low.
HNF TRIAD Academic Partner Connecticut Children’s Publishes Results of CMT Pediatric Natural History Study to Better Understand Ankle Function and Gait HNF-funded research sought to determine how age and CMT type affect ankle strength in children. A detailed...
Hereditary Neuropathy Foundation Re-Launches One-of-A-Kind Patient Registry for Charcot-Marie-Tooth Disease Research
Hereditary Neuropathy Foundation Re-Launches One-of-A-Kind Patient Registry for Charcot-Marie-Tooth Disease Research This user-friendly platform allows patients, caregivers, clinicians, and researchers the ability to manage health data to elevate CMT patient care. The...
Kenneth Raymond provides an overview of why sharing CMT symptoms in GRIN is so important!
Interested in driving CMT research forward? Watch our new 7-min GRIN Demo video explaining the steps in creating and updating your GRIN account!