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Publications by Stephanie C. M. Nijmeijer
Limited Data to Evaluate Real‐World Effectiveness of Enzyme Replacement Therapy for Mucopolysaccharidosis Type I
Journal of Inherited Metabolic Disease
Genetics
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Mucopolysaccharidosis Type II: Enzyme Replacement Therapy Efficiency
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Immune Response to Intrathecal Enzyme Replacement Therapy in Mucopolysaccharidosis I Patients
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Enzyme Replacement Therapy Treatment From Birth Increases Therapeutic Efficacy and Generates Tolerance to Enzyme in Canine Mucopolysaccharidosis Type I
Pharmacoeconomic Evaluation of Drugs Used in Enzyme Replacement Therapy Mucopolysaccharidosis Type II
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Cost-Effectiveness of Enzyme Replacement Therapy for Type 1 Gaucher Disease
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Psy97 - Cost-Effectiveness Analysis of Enzyme Replacement Therapy for Type 1 Gaucher Disease
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Response to Enzyme Replacement Therapy in Patients With Gaucher Disease Type I
Pcp53 - A Diagnostic Framework to Evaluate Real-World Data Sources for Real-World Evidence Generation
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Monitoring Dose Response of Enzyme Replacement Therapy in Feline Mucopolysaccharidosis Type VI by Tandem Mass Spectrometry
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