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Pathogenesis, Epidemiology, Diagnosis and Clinical Aspects of Smith-Lemli-Opitz Syndrome

INTRODUCTION: Smith-Lemli-Opitz Syndrome (SLOS) is a malformation syndrome inherited in an autosomal recessive fashion. It is due to a metabolic defect in the conversion of 7-dehydrocholesterol to cholesterol, which leads to an accumulation of 7-dehydrocholesterol and frequently a deficiency of chol...

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Pubblicato in:Expert Opin Orphan Drugs
Autori principali: Bianconi, Simona E., Cross, Joanna L., Wassif, Christopher A., Porter, Forbes D.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2015
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4343216/
https://ncbi.nlm.nih.gov/pubmed/25734025
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1517/21678707.2015.1014472
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