14 OCTOBER 2025, Seoul/London — A rapidly evolving therapeutic landscape for inherited neuropathies — one in which gene silencing, gene editing, and biomarker-driven innovation are reshaping what is possible for patients once considered untreatable. Speaking at the World Congress of Neurology (WCN), Prof. Mary Reilly MBE, MD, FRCP, FRCPI, FMedSci, Professor of Clinical Neurology and Consultant Neurologist, UCL Queen Square Institute of Neurology, London, shares that science has finally caught up with the vision.
“After decades of mapping the genetic causes of neuropathy, we are finally treating them at their source,” said Reilly. “What once felt theoretical is now entering the clinic.”
Prof. Reilly traced the path from early genetic discoveries in transthyretin (TTR) amyloidosis — once a uniformly fatal condition — to today’s RNA- and CRISPR-based therapies that silence or edit disease-causing genes directly. These breakthroughs have transformed outcomes and set a precedent for applying gene-based treatments to broader classes of Charcot-Marie-Tooth (CMT) and related disorders.
Biomarkers such as neurofilament light chain and MRI fat fraction are emerging as critical tools to measure treatment response and accelerate regulatory approval.
“For slowly progressive neurogenetic diseases, we need surrogate endpoints that predict clinical change,” said Prof. Reilly. “Without them, our trials will take too long, and progress will slow.”
As patients live longer, novel disease patterns, such as central nervous system involvement in TTR amyloidosis, are beginning to emerge, underscoring the need to study the natural history of treated diseases. This calls for collaboration across industry, regulators, and academia to ensure delivery challenges, safety standards, and long trial timelines do not stall innovation.
“We’re learning how to deliver therapies not just to the liver or muscle, but to the peripheral nerve itself,” she said. “Each barrier we overcome expands what’s possible for the next disease.”
The precision tools of modern genetics, once confined to theory, are now rewriting the future of neurology and neurodegenerative care.
“We are at the cusp of a transformative moment,” Prof. Reilly concluded. “Inherited neuropathies are moving from gene discovery to gene therapy. The next challenge — and opportunity — is to make these treatments safe, scalable, and accessible to every patient who needs them.”
About the World Congress of Neurology
The World Federation of Neurology’s World Congress of Neurology brings together leading neuroscientists and public health experts to turn research into action and emphasize the importance of brain health across the globe. The 27th biennial conference was held in Seoul, South Korea from October 12 to 15, 2025, and was co-hosted by the Korean Neurological Association (KNA).
About the World Federation of Neurology
With support from its 126 Member Societies, the World Federation of Neurology fosters quality neurology and brain health worldwide by promoting neurological education and training, emphasizing under-resourced areas of the world. As a non-state actor in official relations, WFN supports the World Health Organization (WHO) efforts to give everyone an equal chance to live a healthy life. With Member Societies around the globe, WFN unites the world’s neurologists to ensure quality neurology and advocate for people to have better brain health. Learn more about the World Federation of Neurology at wfneurology.org.
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