CelluTarget · Multifocal Motor Neuropathy · Research Frontiers
Cellular BiologyPublished Research

iPSC-derived motor neuron models of MMN

Overview
Research Summary

Researchers have built disease models using induced pluripotent stem cell (iPSC)-derived motor neurons exposed to patient serum containing anti-GM1 IgM antibodies. These models reproduce complement activation and structural neuronal damage seen in MMN, giving scientists a human cellular system to study disease mechanism and test candidate therapies (including empasiprubart) without needing nerve biopsy tissue. This is a foundational tool for future target discovery and drug testing in a genuinely human — not just animal — system.

Citations
Sources2 sources
Peer-Reviewed
IgM Anti-Ganglioside Binding and Complement Activation in an iPSC-Derived Motor Neuron Model for Multifocal Motor Neuropathy
Published January 2026
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Peer-Reviewed
Autoantibody pathogenicity in a multifocal motor neuropathy induced pluripotent stem cell-derived model (Harschnitz et al., Ann Neurol 2016;80:71-88)
Published July 2016
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