2025 Global CMT Research Convention

Agenda

The schedule will continute to be updated as additional speakers are confirmed. Please check back for the latest agenda. 

Scientific Program

Thursday, September 25, 2025: 

Check in Begins at 12:00 PM

12:30 PM: Emerging Researcher  Forum

  • For Registered Participants Only

2:30 PM: Welcome

2:35 PM: Genetic Models for CMT

  • Dr. Steven S. Scherer, MD, PhD, Emeritus Professor of Neurology, University of Pennsylvania

3:00 PM: Session: Emerging CMT Drug Development Research Updates

  • Targeting SARM1 and Axon Degeneration as a Design-Modifying Strategy for the Treatment of CMT2A
    • Dr. Bradlee Heckmann, PhD, Co-founder, President, Chief Scientific Officer, Asha Therapeutics
  • Advancing Medicines for Charcot-Marie-Tooth Disease: Development of Antibody Oligonucleotide Conjugates for Peripheral Nerve Delivery
    • Dr. Carlos Loya, PhD, Venture Partner, 82VS

4:00 PM: Break

4:30 PM: Session: Gene Therapy Advancements for CMT

  • AAV Vector Delivery Strategies for Peripheral Nerves
    • Dr. Casey Maguire, PhD, Associate Professor of Neurology, Harvard Medical School,  Investigator, Associate Professor of Neurology, Mass General Research Institute
  • Next-Generation Strategies for Next-Generation Schwann Cell Targeting Therapeutics
    • Dr. Leszek Lisowski, MD, PhD, Unit Head, Translational Vectorology Unit, Children’s Medical Research Institute
  • Intrathecal Gene Replacement for IGHMBP2-related Conditions
    • Dr. Megan Waldrop, MD,  Pediatric Neurologist, Nationwide Children’s Hospital, Assistant Professor of Pediatrics and Neurology, The Ohio State University College of Medicine
  • Advancing Polymer Nanoparticle Delivery of Therapeutic NF1 Signaling Epigenetic Regulatory Protein Payloads to Schwann Cells
    • Dr. Ken Sims, PhD, Senior Materials Scientist, Battelle

6:00 PM: Oral Presentations From Selected Posters

6:30 PM: Welcome Reception + Poster Viewing

Friday, September 26, 2025: 

9:00 AM: Welcome and Pathfinder Award Presentation

9:15 AM: Keynote: From Genes to Treatments: Strategic Considerations for CMT Therapy Development 

  • Dr. Mary Reilly, MD, Professor of Neurology, University College London, Department of Neuromuscular Diseases

10:00 AM: Break

10:30 AM: Session: Innovative Trial Designs for CMT

  • Clinical Trial Design for Gene-Based Therapies in Ultra-Rare Diseases: Lessons from Hereditary Spastic Paraplegia

    • Dr. Darius Ebrahimi-Fakhari, MD, PhD, Director, Movement Disorders Program; Attending, Department of Neurology, Boston Children’s Hospital, Assistant Professor of Neurology, Harvard Medical School

  • Preparing for Clinical Trials in CMT2C: Challenges, Insights, and Unanswered Questions

    • Dr. Brett McCray, MD, PhD, Assistant Professor of Neurology, University of Michigan

  • Dr. Katy J. Eichinger, PhD, DPT, Associate Professor of Neurology, University of Rochester Medical Center

12:00 PM: Lunch + Poster Viewing

1:00 PM: Session: Advancing CMT Research Projects to Industry

  • Dr. Brian Kaspar, PhD, Chief Scientific Officer, Insmed Incorporated

  • Elevating tRNAGly Levels As A Therapeutic Approach For Glycyl-tRNA Synthetase-Associated Peripheral Neuropathy (CMT2D)

    • Dr. Maria Landinez, PhD, Postdoctoral Researcher, Donders Institute for Brain, Cognition, and Behaviour

  • The SARM1 Axon Degeneration Pathway: The Journey from Academic Lab to Small Biotech to Big Pharma

    • Dr. Aaron DiAntonio, MD, PhD, Professor of Developmental Biology, WashU Medicine

2:00 PM: Break and Poster Viewing

2:30 PM: Creative Solutions for Advancing and Commercializing Rare Disease Therapeutics

  • A Journey to Cure Michael: A Parent’s Journey to Cure His Son and Eradicate a Rare Disorder
    • Terry Pirovolakis, Founder and CEO, Elpida Therapeutics

3:00 PM: Oral Presentations From Selected Abstracts

3:45 PM: Break and Poster Viewing

4:15 PM: Funding CMT Research – Insight Into the Current Research Funding Environment and Ways to Overcome Challenges

  • Dr. Walter J. Koroshetz, MD, Director, NIH National Institute of Neurological Disorders and Stroke

4:45 PM: ToPIC: Building a CMT Guidance Document for the FDA

  • Dr. Shannon Strom, PhD, Senior Vice President of Regulatory Affairs, Aerogen Pharma

5:15 PM: Forum: Obtaining Patient Perspectives to Help Inform Therapeutic Development and Clinical Trial Design

6:30 PM: Networking Reception + Poster Viewing

Patient Experience

Friday, September 26, 2025: 

5:00 PM: Forum: Obtaining Patient Perspectives to Help Inform Therapeutic Development and Clinical Trial Design

Saturday, September 27, 2025: 

8:00 AM: Breakfast/Networking With the CMT Community

9:00 AM: Opening Remarks

  • Laura M. MacNeill, CEO, CMTRF
  • Susan Ruediger, Chief Mission Officer, CMTRF
  • Chelsea Layton, Community Engagement Manager, CMTRF

9:40 AM: Concurrent Sessions

What is CMT

  • Dr. Mary Reilly, MD, Professor of Neurology, University College London, Department of Neuromuscular Diseases

Life Cycle and Development of a Drug

  • Dan Brennan, Senior Vice President, Corporate and Commercial Strategy, NMD Pharma

Types of Therapies: Gene Therapy vs. Other Interventions

    10:10 AM: Break

    10:20 AM: Concurrent Sessions

    CMT Type 1 – Mechanisms and Planned Interventions

    • Dr. Mary Reilly, MD, Professor of Neurology, University College London, Department of Neuromuscular Diseases

    CMT Type 2 – Mechanisms and Planned Interventions

    • Dr. Reza Seyedsadjadi, MD, Neurologist, Director, Charcot-Marie-Tooth Center of Excellence, Massachusetts General Hospital, Assistant Professor of Neurology, Harvard Medical School

    CMT Type 4 – Mechanisms and Planned Interventions

    • Dr. Brett McCray, MD, PhD, Assistant Professor of Neurology, University of Michigan

    10:40AM: Break

    10:50 AM: Concurrent Sessions

    CMT Drug Pipeline

    • Robert Prior, PhD, Postdoctoral Researcher,VIB-KU Leuven

    CMT Bracing

    • Ken Cornell, CO, CMT Mobility Rescue

    Unseen Struggles: Navigating Shame, Grief and Uncertainty with CMT

    • Sophia Hilsen, LMHCA, NCC, MAEd, Mental Health Therapist 

    11:20AM: Break 

      11:30 AM: Your Connection to CMT

      12:15 PM: Lunch

      12:45 PM: Fireside Chat With a Person in a CMT Clinical Trial

      1:15 PM: Clinical Trials 101 and How Trials Are Designed Differently for Rare vs. Prevalent Types

      • Jenneen DeFiore, Executive Director, Neuroscience, PPD part of Thermo Fisher Scientific

        1:45 PM: Current and Upcoming Clinical Trials

        • Dan Brennan, Senior Vice President, Corporate and Commercial Strategy, NMD Pharma
        • Dottie Caplan, Senior Vice President, Patient Advocacy & Engagement, Applied Therapeutics
        • David Goldstein, PhD, Co-Founder and CEO, Actio Biosciences

        2:00 PM: Staying Encouraged, Even if You’re Not Eligible

        • Susan Ruediger, Chief Mission Officer, CMTRF

        2:10 PM: Break

        2:20 PM: Recapping ENDGAME and Introducing Velocity

        • Susan Ruediger, Chief Mission Officer, CMTRF
        • Santo Caivano, CMTRF Board of Directors

        2:30 PM: Using Your Voice

        • Chelsea Layton, Community Engagement Manager, CMTRF
        • CMTRF Ambassadors

        3:00 PM: Participating in Natural History and Biomarker Studies

        • Dr. Mary Reilly, MD, Professor of Neurology, University College London, Department of Neuromuscular Diseases

        3:30 PM: Genetic Testing – When, Why and How to Test and Contributing to the Body of Research 

        • Dr. Reza Seyedsadjadi, MD, Neurologist, Director, Charcot-Marie-Tooth Center of Excellence, Massachusetts General Hospital, Assistant Professor of Neurology, Harvard Medical School

        4:00 PM: Wrap Up & Adjourn

        4:15 PM: Zero K: Race to End CMT

        • For registered participants only. Convention attendees can use the promo code 2025CMTRF for free registration. For more information, or to register, visit cmtrf.org/bostonzerok.