Michelle E. Kruijshaar
Rotterdam, ZH, Netherlands
Last Updated: 09/26/2026
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Found 58 publications
Bayesian multivariate linear mixed-effects models with varied association structures.
Journal: Statistical methods in medical research
Published: June 30, 2026
Real-world evidence for Pompe disease remains fragmented. Comment on "A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem" by Klein et al.
Journal: Orphanet journal of rare diseases
Published: August 26, 2024
Start, switch and stop (triple-S) criteria for enzyme replacement therapy of late-onset Pompe disease: European Pompe Consortium recommendation update 2024.
Journal: European journal of neurology
Published: December 04, 2023
Establishing how much improvement in lung function and distance walked is clinically important for adult patients with Pompe disease.
Journal: European journal of neurology
Published: June 29, 2023
Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions.
Journal: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy
Published: May 21, 2023
Association between changes in pulmonary function and in patient reported outcomes during enzyme therapy of adult patients with late-onset Pompe disease.
Journal: Journal of inherited metabolic disease
Published: September 23, 2022
Evaluation of real-life outcome data of patients with spinal muscular atrophy treated with nusinersen in Switzerland.
Journal: Neuromuscular disorders : NMD
Published: October 17, 2021
Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium.
Journal: The Lancet. Child & adolescent health
Published: July 16, 2021
Positive association between physical outcomes and patient-reported outcomes in late-onset Pompe disease: a cross sectional study.
Journal: Orphanet journal of rare diseases
Published: May 04, 2020
Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease.
Journal: Neurology
Published: February 15, 2019
The ACE I/D polymorphism does not explain heterogeneity of natural course and response to enzyme replacement therapy in Pompe disease.
Journal: PloS one
Published: September 12, 2018
Last Updated: 09/26/2026