2025 Global CMT Research Convention sessions

THU · SEP 25 Technical Thursday Welcome Address THU · SEP 25 Technical Genetic Models for CMT Steven S. Scherer THU · SEP 25 Technical Targeting SARM1 and Axon Degeneration as a Design-Modifying Strategy for the Treatment of CMT2A Bradlee Heckmann THU · SEP 25 Technical Targeting Cytosolic HDAC6 Inhibition for the Treatment of CMT and Beyond Gerhard Koenig THU · SEP 25 Technical Advancing Medicines for Charcot-Marie-Tooth Disease Carlos Loya THU · SEP 25 Technical AAV Vector Delivery Strategies for Peripheral Nerves Casey Maguire THU · SEP 25 Technical Intrathecal Gene Replacement for IGHMBP2-related Conditions Megan Waldrop THU · SEP 25 Technical Next-Generation Strategies for Next-Generation Schwann Cell Targeting Therapeutics Leszek Lisowski THU · SEP 25 Technical Advancing Polymer Nanoparticle Delivery Kenneth R. Sims Jr. THU · SEP 25 Technical Small Molecule Modulation of PMP22 Expression and Trafficking THU · SEP 25 Technical 3D Cellular Model of myelination Using Human induced Pluripotent Stem Cells to Study CMT4C FRI · SEP 26 Technical Friday Welcome Address Laura M. MacNeill, Brett A. McCray & Susan Ruediger FRI · SEP 26 Technical Pathfinder Award Presentation Laura M. MacNeill, Brett A. McCray & Susan Ruediger FRI · SEP 26 Technical Keynote: From Genes to Treatments: Strategic Considerations for CMT Therapy Development Mary Reilly FRI · SEP 26 Technical Clinical Trial Design for Gene-Based Therapies in Ultra-Rare Diseases Darius Ebrahimi-Fakhari FRI · SEP 26 Technical Preparing for Clinical Trials in CMT2C: Challenges, Insights, and Unanswered Questions Brett A. McCray FRI · SEP 26 Technical Using Wearable Sensors and Technology to Assess Individuals Living with CMT Katy J. Eichinger FRI · SEP 26 Technical Elevating tRNAGly Levels As A Therapeutic Approach For CMT2D Maria Landinez FRI · SEP 26 Technical From Bench to Bedside: The Development of a Gene Therapy for Spinal Muscular Dystrophy Brian Kaspar FRI · SEP 26 Technical The SARM1 Axon Degeneration Pathway: The Journey from Academic Lab to Small Biotech to Big Pharma Aaron DiAntonio FRI · SEP 26 Technical A Journey to Cure Michael: A Parent’s Journey to Cure His Son and Eradicate a Rare Disorder Terry Pirovolakis FRI · SEP 26 Technical Long-term Exposure to Elevated Sorbitol Levels Leads to Prog. of Neuropathic Symptoms in CMT-SORD FRI · SEP 26 Technical Immune Cell Infiltration and Nanoparticle Targeting in the Connexin32 Knockout Model of CMT1X FRI · SEP 26 Technical A Novel Therapeutic Pipeline for Charcot-Marie-Tooth Disease Type 2A FRI · SEP 26 Technical Funding CMT Research–Insight Into the Current Funding Environment and Ways to Overcome Challenges Walter J. Koroshetz FRI · SEP 26 Technical ToPIC: Building a CMT Guidance Document for the FDA Shannon Strom

FRI · SEP 26 Patient-friendly Forum: Obtaining Patient Perspectives to Help Inform Therapeutic Development & Clinical Trial Design Moderated by Meghan Drummond SAT · SEP 27 Patient-friendly Patient Experience Opening Remarks Susan Ruediger, Chelsea Layton & Laura M. MacNeill SAT · SEP 27 Patient-friendly Catalyst Award Presentation Susan Ruediger, Chelsea Layton & Laura M. MacNeill SAT · SEP 27 Patient-friendly What is CMT Mary Reilly SAT · SEP 27 Patient-friendly CMT Type 1 – Mechanisms and Planned Interventions Mary Reilly SAT · SEP 27 Patient-friendly Drugs Under Development for CMT Robert Prior SAT · SEP 27 Patient-friendly Owning Your Story: How and Why to Share Your Journey With CMT Laura M. MacNeill SAT · SEP 27 Patient-friendly Current and Emerging Clinical Trials for CMT Dan Brennan, Dottie Caplan & David Goldstein SAT · SEP 27 Patient-friendly Understanding Clinical Trials Jenneen DeFiore SAT · SEP 27 Patient-friendly Trials for CMT: One Woman’s Experience Peter J. de Silva & Cara Leath SAT · SEP 27 Patient-friendly When you are not Eligible: Finding Purpose and Progress Beyond Clinical Trials Susan Ruediger SAT · SEP 27 Patient-friendly CMT Progress Fueled by You Susan Ruediger & Santo Caivano SAT · SEP 27 Patient-friendly Speaking Up for CMT: Turning Awareness Into Action Chelsea Layton, Jodi Balog, Ann Fairchild, Joe Hogan & Anushka Noori SAT · SEP 27 Patient-friendly The Power of Participation: Contributing to Research Without Enrolling in a Trial Reza Seyedsadjadi