Learn About Homozygous Familial Hypercholesterolemia (HoFH)

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What are the Latest Advances for Homozygous Familial Hypercholesterolemia (HoFH)?
Lipoprotein apheresis in patients with familial hypercholesterolemia: a single center research.
Is Liver Transplant Curative in Homozygous Familial Hypercholesterolemia? A Review of Nine Global Cases.
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Lessons learned from the evinacumab trials in the treatment of homozygous familial hypercholesterolemia.
What are the latest Homozygous Familial Hypercholesterolemia (HoFH) Clinical Trials?
HoFH, the International Clinical Collaborators - A Global HoFH Data-sharing Platform

Summary: Homozygous familial hypercholesterolemia (HoFH), a rare inherited disorder caused by bi-allelic mutations in the LDL Receptor pathway, is characterized by extremely elevated levels of low-density lipoprotein cholesterol (LDL-C) from birth and premature atherosclerotic cardiovascular disease (ASCVD). Our current knowledge about HoFH is disjointed and largely stems from relatively small case series ...

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Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13)

Summary: This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).