Sen debuterende Pompes sygdom En open- label klinisk undersøgelse vurderede sikkerhed og effekt for Myozyme hos 5 patienter med sen debuterende Pompes sygdom, der var fra 5 til 15 år ved behandlingens start.
9 Late-onset Pompe disease An open-label clinical trial assessed the safety and efficacy of Myozyme in 5 patients with late-onset Pompe disease who ranged in age from 5 to 15 years at initiation of treatment.