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Results from this trial showed that Miplyffa, in combination with miglustat, halted disease progression through 12 months of treatment, as evidenced by a decrease of 0.2 points from baseline on the rescored 4-domain NPC Clinical Severity Scale
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Treatment should be discontinued after 12 weeks on the maintenance dose if patients have not lost at least 5% of their initial body weight
Qi X, Zheng L, Lee K, Kim D, Kim C, Cai D, et al
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