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Treatment of Smith-Lemli-Opitz Syndrome and Other Sterol Disorders

Smith-Lemli-Opitz syndrome (SLOS) is an autosomal recessive genetic condition with a broad phenotype that results from deficiency of the final enzyme of the cholesterol synthesis pathway. This defect causes low or low-normal plasma cholesterol levels and increased 7- and 8-dehydrocholesterol (DHC) l...

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Main Authors: Svoboda, Melissa D., Christie, Jill M., Eroglu, Yasemen, Freeman, Kurt A., Steiner, Robert D.
格式: Artigo
語言:Inglês
出版: 2012
主題:
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3890258/
https://ncbi.nlm.nih.gov/pubmed/23042642
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajmg.c.31347
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