2025 Global CMT Research Convention
Agenda
Scientific Program
Thursday, September 25, 2025:
Young Investigator Forum
- For Registered Participants Only
Break
Welcome
CMT Drug Development: An Overview of Progress, Emerging Advancements and Remaining Hurdles
Session: Emerging CMT Drug Development Research Updates
Break
Session: Gene Therapy Advancements for CMT
Oral Presentations from Selected Posters
Welcome Reception + Poster Viewing
Friday, September 26, 2025:
9:00 AM – Opening Remarks
Keynote: From Genes to Treatments: Strategic Considerations for CMT Therapy Development
- Dr. Mary Reilly, Professor of Neurology, University College London, Department of Neuromuscular Diseases
Break
Session: Innovative Trial Designs for CMT
Lunch + Poster Viewing
Session: Advancing CMT Research Projects to Industry
Creative Solutions for Advancing and Commercializing Rare Disease Therapeutics
Break
Panel: Funding CMT Research – Insight into the Current Research Funding Environment and Ways to Overcome Challenges
Oral Presentations from Selected Abstracts
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
Saturday, September 27, 2025:
8:00 AM – Breakfast
9:00 AM – Opening Remarks
- Laura M. MacNeill, CEO, CMTRF
- Susan Ruediger, Chief Mission Officer, CMTRF
Concurrent Sessions
What is CMT
- Dr. Mary Reilly, 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
Concurrent Sessions
Types of Therapies: Gene Therapy vs. Other Interventions
- Dr. Riann Egusquiza, Director of Research, CMTRF
CMT Type 1 – Mechanisms and Planned Interventions
- Dr. Mary Reilly, Professor of Neurology, University College London, Department of Neuromuscular Diseases
CMT Type 2 – Mechanisms and Planned Interventions
CMT Type 4 – Mechanisms and Planned Interventions
Who Funds CMT Drug Development
How and Why to Talk About Your Connection to CMT
Lunch
Clinical Trials 101
- Jenneen DeFiore, Executive Director, Neuroscience, PPD part of Thermo Fisher Scientific
How Trials are Designed Differently for Rare vs. Prevalent Types
Fireside Chat With a CMT Clinical Trial Patient
How to Enroll in Current and Upcoming Trials
- Dan Brennan, Senior Vice President, Corporate and Commercial Strategy, NMD Pharma
- Dottie Caplan, Senior Vice President, Patient Advocacy & Engagement, Applied Therapeutics
How to Stay Encouraged, Even if You’re Not Eligible
- Susan Ruediger, Chief Mission Officer, CMTRF
Break
How to Use Your Voice
- Chelsea Layton, Community Engagement Manager, CMTRF
Contributing to the Body of Research
Genetic Testing – When, Why and How to Test
Participate in Natural History Studies and Biomarker Studies
- Dr. Mary Reilly, Professor of Neurology, University College London, Department of Neuromuscular Diseases
When and Why to Advocate to State and Federal Government
Wrap Up
- Laura M. MacNeill, CEO, CMTRF
- Susan Ruediger, Chief Mission Officer, CMTRF
Adjourn