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: | , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
MDPI AG
2018-12-01
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| coleção: | Brain Sciences |
| Assuntos: | |
| Acesso em linha: | https://www.mdpi.com/2076-3425/8/12/214 |
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