2026 Global CMT Research Convention sessions

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Keynote: Building the Drug Discovery Engine of the Future with AI-Empowered Nodal Biology

Dr. Anna Greka · Broad Institute of MIT and Harvard

Many seemingly unrelated genetic diseases converge on shared “nodal” pathways — which means the next CMT breakthrough could come from a lab studying something else entirely.

Watch the keynote Browse all 42 talks
FRI · SEP 25

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New to the convention? These are the best first watches.
5 talks
SAT · SEP 26 Patient-friendly Patient Day Keynote: The Momentum Found in CMT Toby Ferguson SAT · SEP 26 Patient-friendly What to Expect in a Clinical Trial Jenneen DeFiore SAT · SEP 26 Patient-friendly The Rest of the Pipeline Dr. Meghan Drummond SAT · SEP 26 Patient-friendly Ways to Be Involved through CMTRF Laura MacNeill SAT · SEP 26 Patient-friendly The Hope I Find in the Progress Made in CMT Patient panel · moderated by Susan Ruediger

Clinical Trials, Explained

From how trials work to what the FDA looks for.
11 talks
SAT · SEP 26 Patient-friendly Updates from Companies NMD Pharma, Actio Biosciences, Elpida Therapeutics, ReviR, EnCell SAT · SEP 26 Patient-friendly What to Expect in a Clinical Trial Jenneen DeFiore SAT · SEP 26 Patient-friendly How I Chose to Participate in a Clinical Trial Paul-Andre Archambalt & Peter DeSilva SAT · SEP 26 Patient-friendly FDA and CMT: How the FDA Thinks About CMT Evaluation Dr. Teresa Buracchio FRI · SEP 25 Patient-friendly Patient Perspectives to Inform Therapeutic Development and Trial Design Patient forum · moderated by Susan Ruediger SAT · SEP 26 Patient-friendly The Reason to Visit an Inherited Neuropathy Consortium Site Dr. Vera Fridman FRI · SEP 25 Technical Clinical Outcome Measures and Biomarkers for CMT: Are We Ready for Clinical Trials? Dr. Vera Fridman FRI · SEP 25 Technical Beyond Patient-Reported Outcomes: Defining Meaningful Clinical Endpoints in CMT Dr. Bopha Chrea FRI · SEP 25 Technical DANCER: A New, Patient-Partnered Platform for Clinical Trials in CMT Dr. Wolfgang Pernice SAT · SEP 26 Patient-friendly Using AI to Build Better Outcome Measurements Dr. Wolfgang Pernice FRI · SEP 25 Technical N-of-1 Personalized Therapy for CMT2S: From Preclinical Models to First-in-Human Dosing Dr. Sandra Smieszek

Gene Therapy & Genetic Medicine

Gene editing, AAV delivery and genetic testing.
8 talks
SAT · SEP 26 Patient-friendly The Pros and Cons of Genetic Testing Dr. Marina Kennerson FRI · SEP 25 Technical AAV Gene Therapy for Peripheral Neuropathies: Progress, Challenges, and the Road Ahead Dr. Rachel Bailey FRI · SEP 25 Technical Overcoming Immune Responses to AAV Vectors Dr. Federico Mingozzi FRI · SEP 25 Technical Clinical Safety and Immunologic Considerations for Systemic AAV Gene Replacement in Duchenne Dr. Diana Bharucha-Goebel THU · SEP 24 Technical Customized CRISPR-Base Editors to Treat Severe Human Diseases Dr. Christiano Alves FRI · SEP 25 Technical In Vivo Testing of CRISPR-Cas9 Gene Therapy Approaches in a Mouse Model of CMT2E Dr. Abigail Tadenev THU · SEP 24 Technical Illuminating the Dark Genome: Genomic Technologies Transforming Gene Discovery Dr. Marina Kennerson THU · SEP 24 Technical Global Strategies to Advance Genomic Medicine Dr. Heidi Rehm

Hearing & CMT

A thread that ran through Thursday and Saturday.
3 talks
SAT · SEP 26 Patient-friendly Hearing and CMT Dr. Ronna Hertzano THU · SEP 24 Technical Overt and Hidden Hearing Loss in CMT Dr. Gabriel Corfas THU · SEP 24 Technical Shared Roads, Different Destinations: Myelinopathy as a Common Thread Between NF2 and CMT Dr. Judith Kempfle

Patient Voices

People living with CMT, in their own words.
4 talks
SAT · SEP 26 Patient-friendly The Hope I Find in the Progress Made in CMT Patient panel · moderated by Susan Ruediger SAT · SEP 26 Patient-friendly How I Chose to Participate in a Clinical Trial Paul-Andre Archambalt & Peter DeSilva FRI · SEP 25 Patient-friendly Patient Perspectives to Inform Therapeutic Development and Trial Design Patient forum · moderated by Susan Ruediger SAT · SEP 26 Patient-friendly Patient Day Keynote: The Momentum Found in CMT Toby Ferguson

Understanding CMT Biology

The disease mechanisms behind every therapy strategy.
6 talks
THU · SEP 24 Technical Humanized CMT1A Model with Myelination Defect for Therapy Development Dr. Gabsang Lee THU · SEP 24 Technical Translational Approaches to CMT1X Dr. Charles Abrams THU · SEP 24 Technical Myelin Membrane Protein Interactions Dr. Robert Piper THU · SEP 24 Technical Peripheral Nerve Homeostasis and Regeneration Dr. Alison Lloyd THU · SEP 24 Technical NODE: A Public Neuropathy Data Portal for Researchers Without Coding Expertise Dr. Carlo Colantuoni THU · SEP 24 Technical Patient-Derived PNS Organoids to Model Diverse CMT1B Phenotypes Mary Kate McCulloch

Brand-New Data

Late-breaking abstracts, selected abstracts and CMTRF-funded early-career work.
11 talks
FRI · SEP 25 Technical Using PMP22 Antisense Therapy to Test Molecular Myelin Architecture in CMT1A Dr. Kathryn Moss FRI · SEP 25 Technical Inhibiting the Integrated Stress Response to Treat tRNA Synthetase–Associated CMT Dr. Timothy Hines FRI · SEP 25 Technical Development of RTX-117, an eIF2B Activator Targeting the Integrated Stress Response Dr. Sridhar Narayan FRI · SEP 25 Technical N-of-1 Personalized Therapy for CMT2S: From Preclinical Models to First-in-Human Dosing Dr. Sandra Smieszek FRI · SEP 25 Technical Inherent Foot Deformities Cause Increased Lateral Column Loading in CMT Tyce Marquez FRI · SEP 25 Technical In Vivo Testing of CRISPR-Cas9 Gene Therapy Approaches in a Mouse Model of CMT2E Dr. Abigail Tadenev FRI · SEP 25 Technical Is Type 1E CMT Much More Common than Recognized? Dr. Chuck Sanders FRI · SEP 25 Technical Automated Sciatic Nerve Fascicle Segmentation in 7T MRI Etash Bhat THU · SEP 24 Technical Engineering AAVs for Schwann Cell Delivery and Blood-Nerve-Barrier Diffusion Daniel Bouch THU · SEP 24 Technical Profiling Neurovascular Cells for Biomarker Discovery Jonathan Alevy THU · SEP 24 Technical Patient-Derived PNS Organoids to Model Diverse CMT1B Phenotypes Mary Kate McCulloch

Funding the Cure

Where the money and the next breakthroughs may come from.
3 talks
FRI · SEP 25 Technical Keynote: Building the Drug Discovery Engine of the Future with AI-Empowered Nodal Biology Dr. Anna Greka FRI · SEP 25 Technical Acquiring Investment Funds from VCs Panel: Biogen, Alloy Therapeutics, NLC Health Ventures, RA Capital SAT · SEP 26 Patient-friendly The Rest of the Pipeline Dr. Meghan Drummond