Which medications are used in the treatment of Smith-Lemli-Opitz syndrome (SLOS)?

Updated: Jan 27, 2021
  • Author: Robert D Steiner, MD; Chief Editor: Luis O Rohena, MD, MS, FAAP, FACMG  more...
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Answer

Supplemental cholesterol may be helpful. Clinical trials are ongoing. Fresh frozen plasma and bile acids have sometimes been administered to patients with Smith-Lemli-Opitz syndrome who have very low plasma cholesterol levels or when mildly to moderately affected patients are unable to take their oral cholesterol supplement, often as a result of illness or surgery.


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