A placebo-controlled trial of simvastatin therapy in Smith-Lemli-Opitz syndrome
Christopher A. Wassif1, Lisa Kratz2, Susan E. Sparks1,3, Courtney Wheeler4, Simona Bianconi1, Andrea Gropman5, Karim A. Calis6, Richard I. Kelley7, Elaine Tierney4, Forbes D. Porter1
1Division of Translational Medicine, Department of Health and Human Services, Section on Molecular Dysmorphology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA
2Department of Neurogenetics, Kennedy Krieger Institute, Baltimore, Maryland, USA
3Current affiliation: Sanofi Genzyme, Cambridge, Massachusetts, USA
4Department of Psychiatry, Kennedy Krieger Institute, Baltimore, Maryland, USA
5Department of Neurology, Children’s National Medical Center, Center for Neuroscience and Behavioral Medicine, Washington, DC, USA
6Department of Health and Human ServicesOffice of the Clinical Director, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA
7Department of Genetics and Genomics, Boston Children’s Hospital, Boston, Massachusetts, USA