Hanka Dekker

Zwolle, OV, Netherlands

Last Updated: 09/26/2026

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Found 14 publications

Safety and efficacy of guanabenz in early-childhood onset vanishing white matter: primary analysis of a single-arm, phase 1/2 trial.

Safety and efficacy of guanabenz in early-childhood onset vanishing white matter: primary analysis of a single-arm, phase 1/2 trial.

Journal: The Lancet. Neurology

Published: May 09, 2026

Consensus-Based Expert Recommendations for Diagnosis and Clinical Management of Vanishing White Matter.

Consensus-Based Expert Recommendations for Diagnosis and Clinical Management of Vanishing White Matter.

Journal: Neurology

Published: November 13, 2025

Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy.

Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy.

Journal: European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society

Published: March 31, 2025

Person-centered outcomes for liver glycogen storage diseases: Development of an international consensus-based standard outcome set.

Person-centered outcomes for liver glycogen storage diseases: Development of an international consensus-based standard outcome set.

Journal: Genetics in medicine : official journal of the American College of Medical Genetics

Published: January 08, 2025

Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases.

Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases.

Journal: Neurology

Published: August 22, 2024

Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management.

Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management.

Journal: European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society

Published: January 26, 2024

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

Core protocol development for phase 2/3 clinical trials in the leukodystrophy vanishing white matter: a consensus statement by the VWM consortium and patient advocates.

Core protocol development for phase 2/3 clinical trials in the leukodystrophy vanishing white matter: a consensus statement by the VWM consortium and patient advocates.

Journal: BMC neurology

Published: January 05, 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

Therapy Trial Design in Vanishing White Matter: An Expert Consortium Opinion.

Therapy Trial Design in Vanishing White Matter: An Expert Consortium Opinion.

Journal: Neurology. Genetics

Published: September 20, 2021

Accelerometer Measured Sedentary and Physical Activity Behaviors of Working Patients after Total Knee Arthroplasty, and their Compensation Between Occupational and Leisure Time.

Accelerometer Measured Sedentary and Physical Activity Behaviors of Working Patients after Total Knee Arthroplasty, and their Compensation Between Occupational and Leisure Time.

Journal: Journal of occupational rehabilitation

Published: September 18, 2020

Consensus guidelines for the diagnosis and management of pyridoxine-dependent epilepsy due to α-aminoadipic semialdehyde dehydrogenase deficiency.

Consensus guidelines for the diagnosis and management of pyridoxine-dependent epilepsy due to α-aminoadipic semialdehyde dehydrogenase deficiency.

Journal: Journal of inherited metabolic disease

Published: August 14, 2020

Showing 1-12 of 14

Last Updated: 09/26/2026