Marion G. Brands

Amsterdam, NH, Netherlands

Last Updated: 09/26/2026

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Found 15 publications

Occurrence of Infusion Associated Reactions and Antidrug Antibodies in Enzyme Replacement Therapy for Fabry Disease and the Effect of Preventive Measures.

Occurrence of Infusion Associated Reactions and Antidrug Antibodies in Enzyme Replacement Therapy for Fabry Disease and the Effect of Preventive Measures.

Journal: Journal of inherited metabolic disease
Published: November 17, 2025

Effectiveness of Pyridoxal-5'-Phosphate in PNPO Deficiency: A Systematic Review.

Effectiveness of Pyridoxal-5'-Phosphate in PNPO Deficiency: A Systematic Review.

Journal: Journal of inherited metabolic disease
Published: May 20, 2025

Treatment Beliefs Reflect Unmet Clinical Needs in Lysosomal Storage Diseases: An Opportunity for a Patient-Centered Approach.

Treatment Beliefs Reflect Unmet Clinical Needs in Lysosomal Storage Diseases: An Opportunity for a Patient-Centered Approach.

Journal: JIMD reports
Published: February 04, 2025

Natural disease course of chronic visceral acid sphingomyelinase deficiency in adults: A first step toward treatment criteria.

Natural disease course of chronic visceral acid sphingomyelinase deficiency in adults: A first step toward treatment criteria.

Journal: Journal of inherited metabolic disease
Published: May 10, 2024

Navigating the outcome maze: a scoping review of outcomes and instruments in clinical trials in genetic neurodevelopmental disorders and intellectual disability.

Navigating the outcome maze: a scoping review of outcomes and instruments in clinical trials in genetic neurodevelopmental disorders and intellectual disability.

Journal: Therapeutic advances in rare disease
Published: October 18, 2023

A Delphi Survey Study to Formulate Statements on the Treatability of Inherited Metabolic Disorders to Decide on Eligibility for Newborn Screening.

A Delphi Survey Study to Formulate Statements on the Treatability of Inherited Metabolic Disorders to Decide on Eligibility for Newborn Screening.

Journal: International journal of neonatal screening
Published: September 10, 2023

Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey.

Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey.

Journal: Orphanet journal of rare diseases
Published: September 05, 2023

Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with tuberous sclerosis complex, mucopolysaccharidosis type III and fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials.

Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with tuberous sclerosis complex, mucopolysaccharidosis type III and fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials.

Journal: BMC psychiatry
Published: July 27, 2023

Glycoprotein non-metastatic protein B (GPNMB) plasma values in patients with chronic visceral acid sphingomyelinase deficiency.

Glycoprotein non-metastatic protein B (GPNMB) plasma values in patients with chronic visceral acid sphingomyelinase deficiency.

Journal: Molecular genetics and metabolism
Published: March 27, 2023

Patients' view on gene therapy development for lysosomal storage disorders: a qualitative study.

Patients' view on gene therapy development for lysosomal storage disorders: a qualitative study.

Journal: Orphanet journal of rare diseases
Published: June 09, 2022

Systematic Review of N-of-1 Studies in Rare Genetic Neurodevelopmental Disorders: The Power of 1.

Systematic Review of N-of-1 Studies in Rare Genetic Neurodevelopmental Disorders: The Power of 1.

Journal: Neurology
Published: August 11, 2020

Long-term cognitive follow-up in children treated for Maroteaux-Lamy syndrome.

Long-term cognitive follow-up in children treated for Maroteaux-Lamy syndrome.

Journal: Journal of inherited metabolic disease
Published: April 25, 2015
Showing 1-12 of 15

Last Updated: 09/26/2026