Publication
Title
Fragile X syndrome neurobiology translates into rational therapy
Author
Abstract
Causal genetic defects have been identified for various neurodevelopmental disorders. A key example in this respect is fragile X syndrome, one of the most frequent genetic causes of intellectual disability and autism. Since the discovery of the causal gene, insights into the underlying pathophysiological mechanisms have increased exponentially. Over the past years, defects were discovered in pathways that are potentially amendable by pharmacological treatment. These findings have inspired the initiation of clinical trials in patients. The targeted pathways converge in part with those of related neurodevelopmental disorders raising hopes that the treatments developed for this specific disorder might be more broadly applicable.
Language
English
Source (journal)
Drug discovery today. - London
Publication
Oxford : Elsevier sci ltd, 2014
ISSN
1359-6446
Volume/pages
19:4(2014), p. 510-519
ISI
000336196500021
Full text (Publisher's DOI)
Full text (publisher's version - intranet only)
UAntwerpen
Faculty/Department
Research group
Publication type
Subject
Affiliation
Publications with a UAntwerp address
External links
Web of Science
Record
Identification
Creation 16.06.2014
Last edited 17.06.2017
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