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