Taste and palatability of Orfadin suspension To determine the acceptability of the suspension in the pediatric population.

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An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1 To evaluate the steady-state exposure to nitisinone during once and twice daily dosing of Orfadin

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A phase 3 clinical study investigating the effects of rhBSSL (an enzyme to help digestion of fat) added to pasteurized breast milk or formula in babies born prematurely The primary objective of this study is to demonstrate that rhBSSL improves growth in preterm infants as compared with placebo when administered in infant formula or PBM.

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To determine a safe and tolerable dose of palifermin in pediatric subjects of different age groups when administered once daily for 3 consecutive days before and 3 consecutive days after a myeloablative chemo-radiotherapy conditioning regimen with allogeneic hematopoietic stem cell transplant (HSCT) To determine a safe and tolerable dose of palifermin in pediatric subjects of different age groups when administered once daily for 3 consecutive days before and 3 consecutive days after a myeloablati...

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A study to evaluate the effects of palifermin in reducing mouth ulceration in subjects with locally advanced head and neck cancer To evaluate the pharmacokinetic profile and biological activity on buccal mucosae of palifermin administered at the dose of 120 μg/kg IV in a cohort of at least 16 (3 palifermin : 1 placebo) locally a...

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