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Literature DB >> 32132878

Statins for Smith-Lemli-Opitz syndrome.

Rami A Ballout¹, Simona Bianconi², Alicia Livinski³, Yi-Ping Fu⁴, Alan T Remaley⁵, Forbes D Porter⁶.

Abstract

This is a protocol for a Cochrane Review (Intervention). The objectives are as follows: 1. To evaluate the efficacy of statin therapy in reducing the frequency or severity of the neurobehavioral abnormalities seen in people with SLOS (e.g. aggression, anxiety, irritability, self-mutilation, autistic behaviors, sleep disturbances, etc.) (Wassif 2017). 2. To evaluate the potential effects of statin therapy on survival.

Entities: Chemical Disease Gene Species

Year: 2020 PMID： 32132878 PMCID： PMC7055734 DOI： 10.1002/14651858.cd013521

Source DB: PubMed Journal: Cochrane Database Syst Rev ISSN： 1361-6137

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References

67 in total

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6. Development and characterization of a hypomorphic Smith-Lemli-Opitz syndrome mouse model and efficacy of simvastatin therapy.

Authors: Lina S Correa-Cerro; Christopher A Wassif; Lisa Kratz; Georgina F Miller; Jeeva P Munasinghe; Alexander Grinberg; Steven J Fliesler; Forbes D Porter
Journal: Hum Mol Genet Date: 2006-01-30 Impact factor: 6.150

Statins for Smith-Lemli-Opitz syndrome.

1. Clinical effects of cholesterol supplementation in six patients with the Smith-Lemli-Opitz syndrome (SLOS)

2. Negative regulation of Hedgehog signaling by the cholesterogenic enzyme 7-dehydrocholesterol reductase.

3. The Aberrant Behavior Checklist-Community: factor validity and effect of subject variables for adults in group homes.

4. Sterol balance in the Smith-Lemli-Opitz syndrome. Reduction in whole body cholesterol synthesis and normal bile acid production.

5. The Children's Sleep Habits Questionnaire (CSHQ): psychometric properties of a survey instrument for school-aged children.

6. Development and characterization of a hypomorphic Smith-Lemli-Opitz syndrome mouse model and efficacy of simvastatin therapy.

Review 7. Treatment of Smith-Lemli-Opitz syndrome and other sterol disorders.

8. The aggression questionnaire.

9. Cytotoxic drug-induced lung disease.

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