您搜索了: pivotalundersøgelse (丹麦语 - 英语)

i en pivotalundersøgelse med 18 patienter blev myozymes farmakokinetiske egenskaber vurderet hos 15 patienter med pompes sygdom med debut i spædbørnsalderen (alle under 6 måneder ved behandlingens start) , der modtog doser på hhv.
in a pivotal trial including 18 patients, the pharmacokinetics of myozyme were evaluated in 15 patients with infantile-onset pompe disease (all aged less than 6 months at treatment-onset) who received doses of 20 mg/kg or 40 mg/kg alglucosidase alfa as an approximate 4 to 6.5-hour infusion, respectively.pharmacokinetics were dose proportional and did not change over time.

den ubehandlede, historiske patientgruppe var i overensstemmelse med pivotalundersøgelsens population og var afledt fra en retrospektiv naturlig anamneseundersøgelse (n=42) hos patienter med pompes sygdom med debut i spædbørnsalderen.
the untreated historical cohort was matched to the pivotal study population and was derived from a retrospective natural history study (n=42) in patients with infantile-onset pompe disease.