Article

Protein-stabilizing and neurotransmission-potentiating activities of a synaptic chaperone modify spinal muscular atrophy in model mice

2026-02-24

Abstract excerpt

Spinal muscular atrophy (SMA) is an oft-fatal infantile-onset neuromuscular disease caused by low SMN protein. Administration of SMN-inducing agents to SMA newborns prevents early mortality, but therapeutic outcomes vary considerably, and disease mechanisms remain poorly understood. Genetic modifiers can provide clues to disease mechanisms and serve as targets for novel treatments. Here, we describe how one such m...

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Literature Corpus work: 05718224-d4c8-5576-afe0-3fdbf45930ab
DOI: 10.64898/2026.02.23.707472

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