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
Breaking News for SORD Deficiency Patients
The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency in the US and Europe.
HNF Contributes CMTRN Data to C-Path’s RDCA-DAP
HNF Contributes CMTRN Data to C-Path’s RDCA-DAP, Strengthening Research Capabilities for Inherited Neuropathies Integration of CMTRN data into RDCA-DAP promises new insights into Charcot-Marie-Tooth disease and related neuropathies, fueling advancements in treatment...
Breaking News: HNF to Host “Revolutionizing Clinical Trials” Panel at Bio 2024
HNF is thrilled to announce that we have been accepted to host a 1-hour panel discussion during the Bio International Convention in San Diego, CA, in June 2024.
CMT Biobank: We Need Your HELP!
HNF is excited to continue collecting samples for researchers and industry to help develop treatments for CMT.
Reducing Genetic Testing Barriers for the Charcot-Marie-Tooth Community
Hereditary Neuropathy Foundation Partners with InformedDNA® on CMT Genie Project
Novartis CMT1A Update
In July 2023, Novartis announced the acquisition of DTx Pharma, a San-Diego based, preclinical stage biotechnology company focused on leveraging its proprietary FALCON platform to develop siRNA therapies for neuroscience indications.
Congratulations to GRIN Advisory Board member, Professor Joshua Burns
Congratulations to GRIN Advisory Board member, Professor Joshua Burns, for being recognized by the Australian Academy of Health and Medical Sciences for his outstanding contributions to medical and health sciences.
Pediatric CMT Natural History Study Enrollment Opening at Two Centers of Excellence
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).
Great News From HNF TRIAD Industry Partner — Pharnext
Pharnext announces new positive results from the ongoing open-label extension study of their drug PXT3003