Title: Developments in treatments for amyotrophic lateral sclerosis via intracerebroventricular or intrathecal delivery
Authors: Van Damme, Philip ×
Robberecht, Wim #
Issue Date: Jul-2014
Series Title: Expert Opinion on Investigational Drugs vol:23 issue:7 pages:955-63
Abstract: Introduction: Amyotrophic lateral scleroses (ALS) are neurodegenerative disorders primarily affecting the motor system. These incurable disorders are relentlessly progressive and typically limit survival to 2 - 5 years after disease onset. An improved knowledge about disease-causing genes, disease proteins and pathways has revealed considerable heterogeneity in ALS. Novel targeted therapies are being developed, but getting these beyond the BBB remains a challenge. Areas covered: The authors review the intracerebroventricular and intrathecal delivery of drugs for the treatment of ALS in preclinical and clinical studies. Expert opinion: Lack of BBB permeability should not hold back the development of promising treatments for ALS, as the available evidence suggest that direct intrathecal or intracerebroventricular administration of drug is a feasible delivery route in patients with ALS.
ISSN: 1354-3784
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Laboratory for Neurobiology
× corresponding author
# (joint) last author

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