A placebo-controlled trial of simvastatin therapy in Smith-Lemli-Opitz syndrome.

Christopher A WassifLisa KratzSusan E SparksCourtney Wheeler Simona Bianconi Andrea L GropmanKarim A CalisRichard I Kelley Elaine Tierney Forbes D Porter

Published in: Genetics in medicine : official journal of the American College of Medical Genetics (2016)

This article reports what is, to our knowledge, the first randomized, placebo-controlled trial designed to test the safety and efficacy of simvastatin therapy in SLOS. Simvastatin seems to be relatively safe in patients with SLOS, improves the serum dehydrocholesterol-to-total sterol ratio, and significantly improves irritability symptoms in patients with mild to classic SLOS.Genet Med 19 3, 297-305.

Keyphrases

double blind
placebo controlled
healthcare
clinical trial
open label
phase iii
case report
randomized controlled trial
stem cells
emergency department
phase ii
physical activity
sleep quality
electronic health record
drug induced
chemotherapy induced