Future multicenter double-blind, randomized, placebo-controlled trials in a large and homogeneous sample of patients should focus on higher doses and more prolonged treatment
In the absence of functional GNE, IGF-1R was hyposialylated and transduced a downstream signal upon IGF-1 (IGF-1R ligand) treatment
Some individuals may experience gastrointestinal discomfort
Barisic I, Balenovic D, Udovicic M, Bardak D, Strinic D, Vlaini J, et al
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Steinberg GR, Hardie DG