← All indications
Smith-Lemli-Opitz Syndrome
An autosomal recessive disorder of CHOLESTEROL metabolism. It is caused by a deficiency of 7-dehydrocholesterol reductase, the enzyme that converts 7-dehydrocholesterol to cholesterol, leading to an abnormally low plasma cholesterol. This syndrome is characterized by multiple CONGENITAL ABNORMALITIES, growth deficiency, and INTELLECTUAL DISABILITY.
Drugs by status
Phase 2 trials (5)
Other trials (1)
Organizations
Organization Involved with Phase 2 Indications (9)
Organization Involved with Other Experimental Indications (2)