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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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Principais autores: Anna W. Lee, Pamela Ventola, Dejan Budimirovic, Elizabeth Berry-Kravis, Jeannie Visootsak
Format: Artigo
Sprog:Inglês
Udgivet: MDPI AG 2018-12-01
Serier:Brain Sciences
Fag:
fragile X syndrome
clinical trials
targeted treatments
drug development
Online adgang:https://www.mdpi.com/2076-3425/8/12/214
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