7 results
The primary objective in the glucocorticoid-continuing subpopulation of men and women treated with chronic glucocorticoid therapy (>= 7.5 mg daily prednisone or its equivalent for >= 3 months and are planning to continue treatment for…
The purpose of this study is to evaluate if microplasmin is safe and effective when injected into the vitreous without performing a vitrectomy.
The objectives of this open-label study are to evaluate the efficacy (GL-3 clearance), pharmacokinetics (PK), and safety parameters (including immunogenicity) for 2 alternative dose regimens of Fabrazyme (0.5 mg/kg every 2 weeks [q2w] and 1.0 mg/kg…
the objective of the current study is to evaluate the safety and efficacy of enzyme therapy in the Dutch population of patients with MPS I, II and VI; to compare this to the natural course of these diseases; to make an inventory of the direct and…
The primary objectives of this study are: * To characterize the effects of 150 mg and 450 mg of AT1001 administered 2 hours before administration of agalsidase on the safety and plasma pharmacokinetics of agalsidase in subjects with Fabry Disease*…
To evaluate the effect of the bisphosphonate risedronate (together with vitamin D and calcium or vitamin D calicum and phosphate in patients with renal phosphate wasting) on bone pain and the evolution of osteolytic bone lesions in patients with FD.
To evaluate the safety and efficacy of PRX-102 compared to agalsidase beta in Fabry disease patients with impaired renal function.