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Recent Patents on Cardiovascular Drug Discovery
ISSN (Print): 1574-8901
ISSN (Online): 2212-3962
VOLUME: 8
ISSUE: 2
DOI: 10.2174/15748901112079990001      Price:  $100









Novel Therapeutic Strategies for the Homozygous Familial Hypercholesterolemia

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Author(s): Giuliana Mombelli and Chiara Pavanello
Pages 143-150 (8)
Abstract:
HoFH is an autosomal co-dominant disease with a prevalence of one in 1,000,000. Mutations of LDL-R gene are responsible for this disease. HoFH needs to be distinguished from autosomal recessive hypercholesterolemia protein (ARH) that causes a similar clinical phenotype. HoFH induces aggressive cardiovascular disease that can develop from birth. These patients possess high LDL-C levels, cutaneous and tendon xanthomas, and accelerated atherosclerosis shown in the first 2 decades of life. Current treatment modalities include life-style modifications, lipid-lowering therapy and LDL-apheresis. However, the treatment goal cannot be achieved only by statin therapy. New therapeutic strategies to lower LDL-C have been developed over recent years. These include monoclonal antibodies binding to PCSK9, inhibition of ApoB production and MTP-inhibitors. This review is focused on new treatments for HoFH and their patents. It is known to be an important contribution in this rare disease, which is difficult to manage.
Keywords:
Current treatments, homozygous familial hypercholesterolemia, mipomersen, MTP inhibitor/Lomitapide, patents, PCSK9 inhibitors.
Affiliation:
Dyslipidemia Center A.O. Ospedale Niguarda Cà Granda, Piazza Ospedale Maggiore 3, 20162, Milan - Italy.