2026 Global CMT Research Convention sessions
Missed Cambridge? Watch every session
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.
FRI · SEP 25
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New to the convention? These are the best first watches.
3 talks
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.
7 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
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
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.
3 talks
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.
2 talks
Scientific Sessions
Research and clinical talks.
11 talks
Technical Welcome and Opening Remarks – 2026 Global CMT Research Convention
Technical Introducing the CMT Research Foundation’s George and Cleary Simpson Award for Emerging Researchers.
Technical AAV-mediated Gene Replacement Therapy for Duchenne Muscular Dystrophy, Dr. Diana Bharucha-Goebel
Technical Realities of Gene Therapy Discussion Forum
Technical Clinical Trial Endpoints Session: Discussion Panel
Technical Clinical Trial Updates from Companies, Dr. David Goldstein (Actio Biosciences, now Setara)
Technical Clinical Trial Updates from Companies, ENCell
Technical Clinical Trial Updates from Companies, Dr. Sridhar Narayan (ReviR)
Technical Many Roads to Treating Charcot-Marie-Tooth Disease, Dr. Meghan Drummond (CMT Research Foundation)
Technical Hearing and CMT, Dr. Ronna Hertzano (University of Maryland / National Institutes of Health)
Technical Closing Remarks from the 2026 Global CMT Research Convention, Laura MacNeill