Efficacy and Safety of Velmanase Alfa in the Treatment of Patients With Alpha-Mannosidosis: Results From the Core and Extension Phase Analysis of a Phase III Multicentre, Double-Blind, Randomised, Placebo-Controlled Trial

Journal of Inherited Metabolic Disease - Netherlands
doi 10.1007/s10545-018-0185-0
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Genetics
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Wiley

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