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Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme.

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…

A Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy

The purpose of the study is to evaluate changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset Pompe disease (LOPD) receiving standard-of-care enzyme replacement therapy (ERT). Additionally,…

DEnosumab for the treatment of FIbrous Dysplasia/McCune-Albright Syndrome in adults (DeFiD): a randomized double-blind placebo-controlled trial

This study has been transitioned to CTIS with ID 2024-511090-30-00 check the CTIS register for the current data. To investigate whether 3 monthly Dmab will improve the clinical, radiological and biochemical manifestations of FD bone lesions.