Ontology highlight
ABSTRACT:
SUBMITTER: van der Veldt N
PROVIDER: S-EPMC7379712 | biostudies-literature | 2019 Feb
REPOSITORIES: biostudies-literature
van der Veldt Nikki N van Rappard Diane F DF van de Pol Laura A LA van der Knaap Marjo S MS van Ouwerkerk Willem J R WJR Becher Jules G JG Wolf Nicole I NI Buizer Annemieke I AI
Developmental medicine and child neurology 20180527 2
Metachromatic leukodystrophy (MLD) is a rare progressive neurological disorder, often accompanied by motor impairments that are challenging to treat. In this case series, we report the course of treatment with intrathecal baclofen (ITB), aimed at improving daily care and comfort in children and young adults with MLD. All patients with MLD in our centre on ITB treatment for a minimum of 6 months were included (n=10; 4 males, 6 females; mean age 10y 8mo [range 6-24y]). Eight patients had MLD with ...[more]