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, and 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

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

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

Creative Solutions for Advancing and Commercializing Rare Disease Therapeutics

Oral Presentations From Selected Abstracts

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

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

CMT Drug Pipeline

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

Concurrent Sessions

Types of Therapies: Gene Therapy vs. Other Interventions

  • Dr. Riann Egusquiza, PhD, Director of Research, CMTRF

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

Funding CMT Drug Development

Your Connection to CMT

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

Fireside Chat With a Person in a CMT Clinical Trial

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

Staying Encouraged, Even if You’re Not Eligible

  • Susan Ruediger, Chief Mission Officer, CMTRF

Using Your Voice

  • Chelsea Layton, Community Engagement Manager, CMTRF

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

Genetic Testing – When, Why and How to Test

Participating in Natural History and Biomarker Studies

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

Advocating to State and Federal Government