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Updated: Jul 31, 2026
07:45
An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy
Published on: October 21, 2014
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Intrathecal baclofen in metachromatic leukodystrophy.
Nikki van der Veldt1, Diane F van Rappard2, Laura A van de Pol2
1Department of Rehabilitation Medicine, VU University Medical Center, Amsterdam Movement Sciences, Amsterdam, the Netherlands.
Developmental Medicine and Child Neurology
|May 29, 2018
Summary
Intrathecal baclofen (ITB) offers a feasible treatment to enhance comfort and daily care for children and young adults with metachromatic leukodystrophy (MLD). MLD patients may require more ITB dose adjustments compared to cerebral palsy patients in the initial treatment phase.
Area of Science:
- Neurology
- Pediatric Neurology
- Movement Disorders
Background:
- Metachromatic leukodystrophy (MLD) is a rare, progressive neurological disorder causing significant motor impairments.
- Managing spasticity and dyskinetic movement disorders in MLD presents therapeutic challenges.
- Intrathecal baclofen (ITB) is an established treatment for spasticity in conditions like cerebral palsy.
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