Looking Beyond Gene Therapy for Treating Charcot-Marie-Tooth Disease: Carla Taveggia, PhD

WATCH TIME: 2 minutes

“These diseases may be genetically very different, but many converge on the same biologic process. If we can restore the proper level of myelination by acting on a common molecular switch, that approach could potentially benefit multiple forms of Charcot-Marie-Tooth disease.”

Despite more than 100 genetic subtypes having been identified, Charcot-Marie-Tooth (CMT) disease remains one of the most challenging inherited neurologic disorders to treat. The remarkable genetic heterogeneity of CMT has complicated traditional drug development, as therapies targeting individual mutations may only benefit small patient populations. Consequently, investigators are increasingly exploring treatment strategies that target shared biologic mechanisms across multiple forms of the disease rather than single-gene defects.

At the 2026 Peripheral Nerve Society (PNS) Annual Meeting in Maastricht, Netherlands, Carla Taveggia, PhD, director of the Division of Neuroscience at IRCCS Istituto Ortopedico Rizzoli, gave a talk on several of the most influential research advances from 2025 and 2026 in CMT and related inherited neuropathies. Among the emerging themes was growing interest in therapeutic approaches that regulate common pathways involved in peripheral nerve myelination, potentially offering broader applicability across genetically distinct neuropathies.

In a conversation with NeurologyLive®, Taveggia discussed why pathway-directed therapies may ultimately prove more practical than mutation-specific gene therapies for many patients with CMT. She explains how insights from basic science and preclinical disease models have identified molecular regulators of myelination that could serve as therapeutic targets across multiple neuropathy subtypes, while also highlighting the continuing role of animal models in advancing translational research.

Click here for more PNS 2026 coverage.