Charcot-Marie-Tooth
8 videos · updated daily
2:59 FDA on Natural History Studies for CMT Trials
The Hereditary Neuropathy Foundation replays an FDA comment from its patient-focused drug development meeting on how natural history studies show what to measure, how often and what patients tolerate in preparing for CMT clinical trials.
55:59 Gene Therapies Demystified: Policy, Access, and the Road Ahead
John Feore, Director of Health Policy & Advocacy for the Institute for Gene Therapies, reviews gene therapy policy at the FDA, HHS and Congress and how patient groups work on access, for the CMT Research Foundation.
53:48 Gene Replacement for CMT2S: Trial Design and Recruitment
Dr. Megan Waldrop of Nationwide Children's Hospital uses the gene replacement trial for CMT2S as a case study for the CMT Research Foundation, covering when evidence supports moving forward, inclusion and exclusion criteria and outcome measures.
32:05 NMD Pharma: SYNAPSE-CMT Phase 2a Results
Thomas Holm Pedersen, CEO of NMD Pharma, presents Phase 2a SYNAPSE-CMT results for ignaseclant (formerly NMD670), reporting gains in grip strength, motor function, walking speed and patient-reported outcomes in CMT1 and CMT2, at the HNF CMT Summit 2026.
1:00:58 Everything You Need to Know About Clinical Trials for CMT
Jenneen DeFiore explains the clinical trial process and common terminology, draws on her family's experience in a CMT clinical trial, and outlines trials underway for Charcot-Marie-Tooth disease as of February 2026, for the CMT Research Foundation.
52:08 Clinical Trial Update from NMD Pharma: CMT Type 1 & Type 2
A clinical study explored whether ignaseclant (formerly NMD670), an investigational drug, could benefit adults with Charcot-Marie-Tooth disease type 1 and type 2.
23:03 CMT-SORD: From Gene Discovery to Clinical Trial
A CMTA Summit session on CMT-SORD, from gene discovery to clinical trial, and the roles of the CMTA, GENESIS, the INC and Applied Therapeutics in developing a treatment.
1:13:23 CMT Drug Development Update: NMD670 and Gene Therapy Delivery
A July 2024 CMT Research Foundation webinar in which Dr. Jorge A. Quiroz of NMD Pharma discusses approval to start a Phase 2 trial of NMD670 in CMT types 1 and 2, and Dr. Carlos Loya of 82VS presents a delivery vehicle for genetic therapies.