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This study affirms the value of the MHFMS in the multicenter pediatric clinical trial setting as a simple, reliable and easy to use outcome measure for documenting gross motor function in children as young as two years of age

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Baric I, Zschocke J, Christensen E, Duran M, Goodman SI, Leonard JV, Muller E, Morton DH, Superti-Furga A, Hoffmann GF: Diagnosis and management of glutaric aciduria type I