Correcting congenital myasthenia-associated acetylcholine receptor defects

Nature, Published online: 01 July 2026; doi:10.1038/s41586-026-10706-1 Cryogenic electron microscopy, chemical biology and electrophysiology are used to determine the structures and functional consequences of representative congenital myasthenic syndrome-associated mutant receptors with and without drugs, uncovering principles of pathogenesis and providing a framework for precision therapies.

Correcting congenital myasthenia-associated acetylcholine receptor defects

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Nature research paper: Correcting congenital myasthenia-associated acetylcholine receptor defects

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