Clinical Development of Targeted Fragile X Syndrome Treatments: An Industry Perspective

Fragile X syndrome (FXS) is the leading known cause of inherited intellectual disability and autism spectrum disorder. It is caused by a mutation of the fragile X mental retardation 1 (<i>FMR1</i>) gene, resulting in a deficit of fragile X mental retardation protein (FMRP). The clinical presentation...

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Autors principals:	Anna W. Lee, Pamela Ventola, Dejan Budimirovic, Elizabeth Berry-Kravis, Jeannie Visootsak
Format:	Artigo
Idioma:	Inglês
Publicat:	MDPI AG 2018-12-01
Col·lecció:	Brain Sciences
Matèries:	fragile X syndrome clinical trials targeted treatments drug development
Accés en línia:	https://www.mdpi.com/2076-3425/8/12/214
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