The first session of the CMT Research Foundation’s Gene Therapies Demystified series gave attendees the building blocks: what gene therapy is, why CMT is a compelling target, and what the science suggests about its potential. Now, the series moves from the foundational to the practical, and the questions get harder.
The second installment, Gene Replacement for CMT2S: Challenges in Trial Design and Recruitment, takes a close look at what it actually takes to bring a gene therapy out of pre-clinical models and into a human study. Using the ongoing gene replacement trial for CMT2S as a case study, this session pulls back the curtain on one of the most consequential and least visible stages of the research process: clinical trial design.
Dr. Megan Waldrop, a pediatric neurologist at Nationwide Children’s Hospital and assistant professor of Pediatrics and Neurology at The Ohio State University College of Medicine, will guide attendees through the scientific and practical challenges researchers face when translating a promising therapy into a study that is rigorous, safe, and meaningful. That includes knowing when the evidence is strong enough to move forward, navigating the difficult decisions behind inclusion and exclusion criteria, and selecting outcome measures that can genuinely detect whether a therapy is working.
These are not abstract questions. Every choice made in trial design has real consequences for the patients who participate, the data that gets collected, and the field’s ability to learn from the results. For anyone following the progress of gene therapies for CMT, understanding how these decisions get made is essential context.
Whether you are a patient, caregiver, clinician, or researcher, this session offers a rare behind-the-scenes perspective on the work that happens long before a therapy reaches anyone’s hands.
The second installment of Gene Therapies Demystified takes place on June 18 at 7pm Eastern.
