Evinacumab for pediatric patients with homozygous familial hypercholesterolemia

A Wiegman, S Greber-Platzer, S Ali, MD Reijman… - Circulation, 2024 - Am Heart Assoc
BACKGROUND: Homozygous familial hypercholesterolemia (HoFH) is a rare genetic
disorder characterized by severely elevated low-density lipoprotein cholesterol (LDL-C) …

[HTML][HTML] Closing the gaps in care of dyslipidemia: Revolutionizing management with digital health and innovative care models

SJ Apple, R Clark, J Daich, ML Gonzalez… - Reviews in …, 2023 - ncbi.nlm.nih.gov
Although great progress has been made in the diagnostic and treatment options for
dyslipidemias, unawareness, underdiagnosis and undertreatment of these disorders remain …

Newborn screening for lipid disorders

X Shao, R Steiner, AL Peterson - Current Opinion in Lipidology, 2024 - journals.lww.com
Historically, newborn screening for lipid disorders was not done for many reasons, but new
research has developed testing methods that may successfully identify common and rare …

Contemporary lipid-lowering management and risk of cardiovascular events in homozygous familial hypercholesterolaemia: insights from the Italian LIPIGEN Registry

L D'Erasmo, S Bini, M Casula, M Gazzotti… - European Journal of …, 2024 - academic.oup.com
Aims The availability of novel lipid-lowering therapies (LLTs) has remarkably changed the
clinical management of homozygous familial hypercholesterolaemia (HoFH). The impact of …

Long-term outcomes of liver transplantation for homozygous familial hypercholesterolaemia in Australia and New Zealand

MM Page, W Hardikar, G Alex, S Bates, S Srinivasan… - Atherosclerosis, 2023 - Elsevier
Background and aims Homozygous familial hypercholesterolaemia (FH) causes severe
cardiovascular disease from childhood. Conventional drug therapy is usually ineffective; …

Universal paediatric screening for familial hypercholesterolaemia

MP McGowan, M Cuchel - The Lancet, 2024 - thelancet.com
Familial hypercholesterolaemia is a common, autosomal, semidominant genetic disorder
that is characterised by increased LDL cholesterol (LDL-C) from birth and, if left untreated …

A machine-learning algorithm using claims data to identify patients with homozygous familial hypercholesterolemia

J Gu, M Epland, X Ma, J Park, RJ Sanchez, Y Li - Scientific Reports, 2024 - nature.com
Homozygous familial hypercholesterolemia (HoFH) is an underdiagnosed and undertreated
ultra-rare disease. We utilized claims data from the Komodo Healthcare Map database to …

Evinacumab: Mechanism of action, clinical, and translational science

R Dingman, S Bihorel, V Gusarova… - Clinical and …, 2024 - Wiley Online Library
Homozygous familial hypercholesterolemia (HoFH) is a rare and serious genetic condition
characterized by premature cardiovascular disease due to severely elevated low‐density …

USPSTF Recommendation on Screening for Lipid Disorders in Children and Adolescents

MP McGowan, M Cuchel, PC Lee - JAMA, 2023 - jamanetwork.com
To the Editor The Agency for Healthcare Research and Quality describes the USPSTF as
follows:“The USPSTF works to improve the health of people nationwide by making …

[HTML][HTML] It is Time to Screen for Homozygous Familial Hypercholesterolemia in the United States

SS Gidding, CM Ballantyne, M Cuchel, S de Ferranti… - Global Heart, 2024 - ncbi.nlm.nih.gov
Homozygous familial hypercholesterolemia (HoFH) is an ultra-rare inherited condition that
affects approximately one in 300,000 people. The disorder is characterized by extremely …