The Peripheral Nerve Society hosted its annual conference in the Netherlands this June, and our research team went to learn about the latest advances, network with field leaders and accelerate progress for CMT treatments and cures.
Here are our scientific team’s takeaways from the 2026 meeting.
The Treatment Pipeline Is Maturing Fast
The breadth and pace of therapeutic development for CMT is striking. Across the conference, researchers presented advances spanning gene therapy, gene editing, RNA-based strategies, and small molecule-based approaches, with multiple programs showing efficacy in pre-clinical models and a growing number nearing human trials.
A recurring and encouraging theme was the potential for strategies that could address multiple CMT subtypes rather than a single genetic cause. The Integrated Stress Response (ISR), a cellular pathway activated under conditions of biological stress, is emerging as one such shared mechanism. A drug already in human trials for another condition showed meaningful benefit across several pre-clinical models of tRNA synthetase-related CMT, including in late-stage disease. The breadth of that signal across related subtypes suggests the ISR may represent a genuine convergence point for therapeutic intervention, and our team is following up directly with the researchers involved.
Gene therapy approaches are also advancing rapidly, with researchers actively engineering next-generation delivery vectors designed to reach Schwann cells with much greater efficiency. Separately, a tRNA overexpression strategy restored function in pre-clinical models of CMT2D even after symptom onset, which is an important proof of concept for the idea that meaningful intervention may be possible well into the disease course.
A particularly creative small molecule approach led by Claire Jacobs used a transdermal patch to deliver low doses of an HDAC2 activator, restoring myelination in a CMT1A pre-clinical model.
Finally, studies by Anne-Sophie Lia showed that stop codon read-through therapy restored production of a key disease-relevant protein in CMT4C cells. Because premature stop codons occur across a large proportion of CMT-associated genes, this approach could serve as a broadly applicable platform for multiple subtypes simultaneously.
Biomarkers: Incremental Progress Toward a High Bar
The field is increasingly aware that neurofilament light chain (NfL), while useful, is not the biomarker CMT needs long term. It lacks specificity, misses small fiber involvement, and does not reliably track progression in slowly evolving disease. Several alternatives were presented, including imaging-based muscle fat fraction, which showed strong validation data in CMT1A, and a serum protein with greater disease specificity than NfL, though its relationship to clinical severity remains unclear.
The assessment from the field is that the next generation of biomarkers will likely require integrating multiple signals, and that AI-driven analysis of large-scale biological datasets is potentially a path to getting there. We see a real opportunity to contribute to this effort.
The overall message from this meeting was one of momentum.
The science is maturing, the community is coordinated, and the pipeline is more substantive than it has ever been.
