Therapeutic Effects of Nizubaglustat in a Mouse Model of GM2 Gangliosidosis.

J Inherit Metab Dis · 2026 · PMC12779495 · PMID 41500827

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just missing significanceno p-value reported

While just missing significance, the trend in the data suggests that cerebellar Purkinje cells are likely spared to a greater extent in nizubaglustat treated mice compared to vehicle treated mice.

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