?Zhang H, Xiong H, Wei C, Yi M, Che Y, Zhuo J, Li X. Evaluation of the safety and efficacy of miglustat for the treatment of Chinese patients with Niemann-Pick disease type C: A prospective, open-label, single-arm, phase IV trial. Intractable Rare Dis Res. 2024 Nov 30;13(4):227-235. doi: 10.5582/irdr.2024.01056. PMID: 39628618; PMCID: PMC11609035.
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