CMT Research Foundation
The CMT Research Foundation has one mission: to find treatments and cures for all types of CMT. We do this by investing in the most promising, translational research. View our videos to learn more about the science and current field of CMT research.
Величайший дар: лекарство от болезни Шарко-Миттала
Величайший дар: Шайла Бозарт
Может ли удаление SARM1 привести к терапии пациентов с синдромом ШМТ 2А?
Trials for CMT: One Woman’s Experience
Current and Emerging Clinical Trials for CMT
Understanding Clinical Trials
When you are not Eligible: Finding Purpose and Progress Beyond Clinical Trials
CMT Progress Fueled by You
Speaking Up for CMT: Turning Awareness Into Action
Thursday Welcome Address
Genetic Models for CMT
Targeting SARM1 and Axon Degeneration as a Design-Modifying Strategy for the Treatment of CMT2A
Targeting Cytosolic HDAC6 Inhibition for the Treatment of CMT and Beyond
Advancing Medicines for Charcot-Marie-Tooth Disease
AAV Vector Delivery Strategies for Peripheral Nerves
Intrathecal Gene Replacement for IGHMBP2-related Conditions
Next-Generation Strategies for Next-Generation Schwann Cell Targeting Therapeutics
Advancing Polymer Nanoparticle Delivery
Small Molecule Modulation of PMP22 Expression and Trafficking
3D Cellular Model of myelination Using Human induced Pluripotent Stem Cells to Study CMT4C
Friday Welcome Address
Pathfinder Award Presentation
Keynote: From Genes to Treatments: Strategic Considerations for CMT Therapy Development
Clinical Trial Design for Gene-Based Therapies in Ultra-Rare Diseases
Preparing for Clinical Trials in CMT2C: Challenges, Insights, and Unanswered Questions
Using Wearable Sensors and Technology to Assess Individuals Living with CMT
Elevating tRNAGly Levels As A Therapeutic Approach For CMT2D
From Bench to Bedside: The Development of a Gene Therapy for Spinal Muscular Dystrophy
The SARM1 Axon Degeneration Pathway: The Journey from Academic Lab to Small Biotech to Big Pharma