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Gaucher disease A primary, autosomal recessive lysosomal storage disease caused by mutations in the GBA1 gene, which encodes -glucocerebrosidase (GCase)

doses were escalated after 5 days from 5 to 10 mg in all multiple-dose trials except in the dosing conditions trial (NCT01572753) [15], in which 10 mg was dosed for 10 days