title: Fragile X syndrome: an update on developing treatment modalities.
abstract: Intellectual disability (ID; mental retardation) is considered an immutable condition. Current medical practices are aimed at relieving symptoms and not at altering the underlying cognitive deficits. Scientific advancements from the past decade have led to the exciting possibility that ID may now be treatable. Moreover, pharmaceutical therapies targeting the most common form of inherited ID, Fragile X syndrome (FXS), may become the new benchmark for central nervous system (CNS) drug discovery: seeking cures for neurodevelopmental disorders.
authors.affiliation: Seaside Therapeutics, 840 Memorial Drive, Cambridge, Masssachusetts 02139, USA.
authors.forename: Aileen
authors.initials: A
authors.lastname: Healy
authors.forename: Roger
authors.initials: R
authors.lastname: Rush
authors.forename: Timothy
authors.initials: T
authors.lastname: Ocain
mesh_terms: D000494:Allosteric Regulation / Q000187:drug effects / Q000502:physiology; D000818:Animals; D001418:Baclofen / Q000031:analogs & derivatives / Q000737:chemistry / Q000494:pharmacology / Q000627:therapeutic use; D002491:Central Nervous System Agents / Q000737:chemistry* / Q000494:pharmacology / Q000627:therapeutic use*; D055808:Drug Discovery / Q000639:trends*; D005600:Fragile X Syndrome / Q000188:drug therapy* / Q000378:metabolism / Q000523:psychology; D006801:Humans; D064529:Receptor, Metabotropic Glutamate 5; D018094:Receptors, Metabotropic Glutamate / Q000819:agonists / Q000037:antagonists & inhibitors / Q000502:physiology*; D016896:Treatment Outcome
chemical_list: D002491:Central Nervous System Agents; D064529:Receptor, Metabotropic Glutamate 5; D018094:Receptors, Metabotropic Glutamate; D001418:Baclofen; C543531:arbaclofen placarbil