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

Young Investigator Forum

  • For Registered Participants Only

CMT Drug Development: An Overview of Progress, Emerging Advancements and Remaining Hurdles

Session: Emerging CMT Drug Development Research Updates

  • Dr. Bradlee Heckmann, PhD, Co-founder, President, Chief Scientific Officer, Asha Therapeutics
  • Dr. Carlos Loya, PhD, Venture Partner, 82VS

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
  • Leszek Lisowski, MD, PhD, Unit Head, Translational Vectorology Unit, Children’s Medical Research Institute
  • Megan Waldrop, MD,  Pediatric Neurologist, Nationwide Children’s Hospital, Assistant Professor of Pediatrics and Neurology, The Ohio State University College of Medicine

Oral Presentations From Selected Posters

Welcome Reception + Poster Viewing

Friday, September 26, 2025: 

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

Session: Innovative Trial Designs for CMT

  • 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
  • Dr. Brett McCray, MD, PhD, Assistant Professor of Neurology, University of Michigan
  • Katy J. Eichinger, PhD, DPT, Associate Professor of Neurology, University of Rochester Medical Center

Lunch + Poster Viewing

Session: Advancing CMT Research Projects to Industry

  • Dr. Brian Kaspar, PhD, Chief Scientific Officer, Insmed Incorporated
  • Dr. Ljudmila Katchan, PhD, Research & Development Lead, NLC Health Ventures
  • The SARM1 Axon Degeneration Pathway: The Journey from Academic Lab to Small Biotech to Big Pharma
    • Aaron DiAntonio, MD, PhD, Professor of Developmental Biology, WashU Medicine

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

Oral Presentations From Selected Abstracts

Funding CMT Research – Insight Into the Current Research Funding Environment and Ways to Overcome Challenges

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

ToPIC: Building a CMT Guidance Document for the FDA

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

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

Networking Reception + Poster Viewing

Patient Experience

Friday, September 26, 2025: 

5:00 PM – Make Your Voice Heard to Researchers

Saturday, September 27, 2025: 

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

9:00 AM – Opening Remarks

  • Laura M. MacNeill, CEO, CMTRF

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

    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

    Concurrent Sessions

    CMT Drug Pipeline

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

      Your Connection to CMT

      Fireside Chat With a Person in a CMT Clinical Trial

      Clinical Trials 101

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

      How Trials Are Designed Differently for Rare vs. Prevalent Types

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

      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

      Staying Encouraged, Even if You’re Not Eligible

      • Susan Ruediger, Chief Mission Officer, CMTRF

      Recapping ENDGAME and Introducing Velocity

      • Susan Ruediger, Chief Mission Officer, CMTRF

      Using Your Voice

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

      Participating in Natural History and Biomarker Studies

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

      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