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A Placebo-Controlled Trial of Simvastatin Therapy in Smith-Lemli-Opitz Syndrome

BACKGROUND: Smith-Lemli-Opitz syndrome (SLOS) is a multiple malformation/cognitive impairment syndrome characterized by the accumulation of 7-dehydrocholesterol (7DHC), a precursor sterol of cholesterol. Simvastatin, an HMG-CoA reductase inhibitor that crosses the blood-brain-barrier, has been propo...

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Detalles Bibliográficos
Autores principales:	Wassif, Christopher A., Kratz, Lisa, Sparks, Susan E., Wheeler, Courtney, Bianconi, Simona, Gropman, Andrea, Calis, Karim A., Kelley, Richard I., Tierney, Elaine, Porter, Forbes D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2016
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5303568/ https://www.ncbi.nlm.nih.gov/pubmed/27513191 http://dx.doi.org/10.1038/gim.2016.102

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