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 (FMR1) gene, resulting in a deficit of fragile X mental retardation protein (FMRP). The clinical presentation of FXS...

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Detalles Bibliográficos
Autores principales: Lee, Anna W., Ventola, Pamela, Budimirovic, Dejan, Berry-Kravis, Elizabeth, Visootsak, Jeannie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2018
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6315847/
https://www.ncbi.nlm.nih.gov/pubmed/30563047
http://dx.doi.org/10.3390/brainsci8120214

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