2024 Global CMT Research Convention sessions
Watch every session from the 2024 convention
Patient Voices: How Your Scientific Progress Provides Hope
Adrienne Weinstein · CMTRF Endgame Campaign Committee
From the 2024 Global CMT Research Convention.
FRI · SEP 27
How would you like to catch up?
Scientific Sessions
Research and clinical talks.
15 talks
FRI · SEP 27
Technical Patient Voices: How Your Scientific Progress Provides Hope
Adrienne Weinstein
FRI · SEP 27
Technical Patient Voices – Living With CMT
Eloise Schlafly
FRI · SEP 27
Technical TRPV4 Inhibition in the Treatment of CMT2C
David Goldstein
FRI · SEP 27
Technical Supporting Translation of CMT1A Gene Therapy Through AAV-mediated Silencing of PMP22
Scott Harper
FRI · SEP 27
Technical Development of Gene Replacement Therapy for CMT4A Demyelinating Neuropathy
Xin Chen
FRI · SEP 27
Technical The Development of Tissue Tropic AAVs – Considerations Inside Out
Guangping Gao
FRI · SEP 27
Technical Developing Lipid Nanoparticles for Non-Liver RNA Delivery
Ryan Zenhausern
FRI · SEP 27
Technical Current Status of Clinical Outcome Assessments and Biomarkers in CMT
Brett McCray
FRI · SEP 27
Technical Understanding the Criteria For Pursuing a Drug Program for CMT
CEO panel moderated by Arthur Suckow
FRI · SEP 27
Technical Discovery of Small Molecules that Alter the Trafficking of PMP22 as A Potential Route
Charles Sanders
FRI · SEP 27
Technical Developing Best-in-class HDAC6 Inhibitors for the Treatment of CMT1A
Frederik Rombouts
FRI · SEP 27
Technical FDA Efforts to Advance Medical Product Development for Rare Diseases
Celia Witten
FRI · SEP 27
Technical Govorestat for CMT-SORD: Designing a Clinical Trial for a Slow Progressing CMT
Riccardo Perfetti
FRI · SEP 27
Technical Understanding Basic Biological Mechanisms to Identify Therapeutic Targets in CMT
Rob Burgess
FRI · SEP 27
Technical Clinical Development of CLC- Inhibitor for the Treatment of Charcot-Marie-Tooth Disease
Jorge A. Quiroz
Patient Experience Sessions
Plain-language talks for people living with CMT and their families.
13 talks
SAT · SEP 28
Patient-friendly You Are UNSTOPPABLE
Chelsea Layton, Gary Berg, John Neville, Lily Sander & Jay Mulrooney
SAT · SEP 28
Patient-friendly Development of Gene Therapy for Rare Diseases
SAT · SEP 28
Patient-friendly Clinical Development of CLC-1 Inhibitors For the Treatment of CMT
SAT · SEP 28
Patient-friendly You and the Future of Clinical Trials in CMT
Panel moderated by Susan Ruediger
SAT · SEP 28
Patient-friendly A Guide to Clinical Research: One Family’s Journey
SAT · SEP 28
Patient-friendly Fueling Progress With ENDGAME – The Campaign to End CMT1A
Peter deSilva, Susan Ruediger & Riann Egusquiza
SAT · SEP 28
Patient-friendly Lessons Learned From SOD1 ALS Clinical Development: Opportunities for CMT Drug Development
SAT · SEP 28
Patient-friendly The State of Drug Development for CMT
Robert Prior & Toby Ferguson
SAT · SEP 28
Patient-friendly Living With CMT
SAT · SEP 28
Patient-friendly What is Happening to my Nerves and Body
Bruce D. Carter
SAT · SEP 28
Patient-friendly Living With CMT
SAT · SEP 28
Patient-friendly UNSTOPPABLE
Chelsea Layton, Gary Berg, John Neville, Lily Sander & Jay Mulrooney
SAT · SEP 28
Patient-friendly Opening/Patients Taking Action
Cleary Simpson, Peter deSilva & Jamal Hill