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A placebo-controlled trial of simvastatin therapy in Smith-Lemli-Opitz syndrome.
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.
Afiliação
  • Wassif CA; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland, USA.
  • Kratz L; Department of Neurogenetics, Kennedy Krieger Institute, Baltimore, Maryland, USA.
  • Sparks SE; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland, USA.
  • Wheeler C; Current affiliation: Sanofi Genzyme, Cambridge, Massachusetts, USA.
  • Bianconi S; Department of Psychiatry, Kennedy Krieger Institute, Baltimore, Maryland, USA.
  • Gropman A; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland, USA.
  • Calis KA; Department of Neurology, Children's National Medical Center, Center for Neuroscience and Behavioral Medicine, Washington, DC, USA.
  • Kelley RI; Office of the Clinical Director, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland, USA.
  • Tierney E; Department of Genetics and Genomics, Boston Children's Hospital, Boston, Massachusetts, USA.
  • Porter FD; Department of Psychiatry, Kennedy Krieger Institute, Baltimore, Maryland, USA.
Genet Med ; 19(3): 297-305, 2017 03.
Article em En | MEDLINE | ID: mdl-27513191

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Smith-Lemli-Opitz / Sinvastatina Tipo de estudo: Clinical_trials Limite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: Genet Med Ano de publicação: 2017 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Smith-Lemli-Opitz / Sinvastatina Tipo de estudo: Clinical_trials Limite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: Genet Med Ano de publicação: 2017 Tipo de documento: Article