The Muscular Dystrophy Association Clinical & Scientific Conference in mid-March is one of the premier gatherings in the neuromuscular disease world, bringing together researchers, clinicians, industry, and advocates to share the latest science. Our research staff was there all week. Here’s what stood out regarding Charcot-Marie-Tooth disease. 

1. Clinical trial readiness is front and center. A dedicated session asked directly: are we ready for CMT clinical trials? From biomarkers to outcome measures from wearables tracking gait and balance, the infrastructure to support drug development is being built right now.

2. Ignaseclant showed efficacy in CMT patients. Top-line safety and efficacy data from the Phase 2a SYNAPSE-CMT trial, which we previously highlighted in a webinar, were presented at the conference. Notably, secondary endpoints for hand strength and dexterity showed sustained improvement — a significant signal for a disease with no approved disease-modifying therapies.

3. Gene therapy for rare CMT subtypes is becoming a clinical reality. Two presentations focused on gene therapy for CMT2S/SMARD1, and what was once a distant horizon is now entering the clinic. Critically, the lessons learned from these programs will inform future gene therapy development across other CMT subtypes.

4. You can’t treat what you can’t measure. Wearable sensors, MRI biomarkers, and digital measures kept surfacing across sessions as essential to running trials that can actually demonstrate meaningful change. Better tools equal better trials. CMTRF recently invested in a project addressing this need specifically.

5. Patient advocacy is driving the science. The MDA Donavon Decker Community Impact in Research Award went to Allison Moore of the Hereditary Neuropathy Foundation — a powerful reminder that the CMT community’s voice is actively shaping the research agenda.