Marieke Biegstraaten
Amsterdam, NH, Netherlands
Last Updated: 09/26/2026
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Found 35 publications
Oncologic orphan drugs approved in the EU - do clinical trial data correspond with real-world effectiveness?
Journal: Orphanet journal of rare diseases
Published: December 08, 2017
Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study.
Journal: Journal of medical genetics
Published: June 12, 2017
Phenotype, disease severity and pain are major determinants of quality of life in Fabry disease: results from a large multicenter cohort study.
Journal: Journal of inherited metabolic disease
Published: May 18, 2017
Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease.
Journal: Molecular genetics and metabolism
Published: March 18, 2017
Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease.
Journal: Blood cells, molecules & diseases
Published: September 20, 2016
Characterization of Classical and Nonclassical Fabry Disease: A Multicenter Study.
Journal: Journal of the American Society of Nephrology : JASN
Published: September 10, 2016
Hearing loss in children with Fabry disease.
Journal: Journal of inherited metabolic disease
Published: June 02, 2016
Erratum to: Pain management strategies for neuropathic pain in Fabry disease - a systematic review.
Journal: BMC neurology
Published: May 18, 2016
Biceps Rerouting after Forearm Osteotomy: An Effective Treatment Strategy for Severe Supination Deformity in Obstetric Plexus Palsy.
Journal: Journal of hand and microsurgery
Published: May 10, 2016
Post-authorisation assessment of orphan drugs.
Journal: Lancet (London, England)
Published: February 05, 2016
Last Updated: 09/26/2026