ホモ接合体抗コレステロール血症治療薬： mipomersen と Lomitapide

アンチセンスオリゴ核酸阻害剤　mipomersen（ antisense oligonucleotide inhibitor ；Gezyme社製）はFHに対しFDA承認されている。

 http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/EndocrinologicandMetabolicDrugsAdvisoryCommittee/UCM323927.pdf

Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Subjects With Coronary Artery Disease (RADICHOL II) http://clinicaltrials.gov/ct2/show/results/NCT00706849

Lomitapide（ microsomal triglyceride transfer protein inhibitor ） も作用機序違うが、承認
http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/DrugsEndocrinologicandMetabolicDrugsAdvisoryCommittee/UCM323841.pdf 

こちらは、Lancetに論文報告

Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study
The Lancet, Early Online Publication, 2 November 2012 doi:10.1016/S0140-6736(12)61731-0