Marjo V. D. K.
- Country
- The Netherlands
- Department
- Child neurology
Research Overview
- Evaluation of ION373 Efficacy and Safety in Alexander Disease: A Phase 1-3 Double-Blind, Randomized, Placebo-Controlled Study
- Evaluation of Fosigotifator Sodium Tromethamine in Adult and Pediatric Patients with Vanishing White Matter Disease: A Phase 1b/2 Open-Label Study
- Evaluation of Safety, Tolerability, and Pharmacokinetics of Guanabenz Acetate in Pediatric Patients with Early-Childhood Onset Vanishing White Matter
Data & Insights
Has run more than one trial at 1 of 3 partner sites.
Specializations
Rare Neurological Disorders
This investigator specializes in treating and researching rare neurological conditions, with particular focus on leukodystrophies and related disorders that affect the brain's white matter.
- Vanishing White Matter Disease (VWM)
- Alexander Disease
The investigator's work centers on innovative therapeutic approaches for these rare neurological conditions, including both novel pharmaceutical compounds and specialized treatment methods.
Therapeutic Innovation
The investigator explores targeted therapeutic interventions for rare neurological disorders, including:
- Intrathecal drug administration
- Novel compound development
- Motor function improvement strategies
Their research encompasses evaluating new therapeutic agents and their effects on neurological function and disease progression.
Clinical Assessment
The investigator's expertise includes comprehensive evaluation of:
- Safety and tolerability profiles
- Motor function assessment
- Disease progression monitoring
Their work involves detailed assessment of therapeutic interventions in pediatric and adult patients with rare neurological conditions.
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Researchers in similar fields or departments
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