|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Microsomal triglyceride transfer protein inhibitor (lomitapide) efficacy in the treatment of patients with homozygous familial hypercholesterolaemia.
|
31403880 |
2020 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Lomitapide is an oral inhibitor of the microsomal triglyceride transfer protein used to treat homozygous familial hypercholesterolemia (HoFH); patients require a low-fat diet to minimize gastrointestinal adverse effects and dietary supplements to prevent nutrient deficiencies.
|
29899183 |
2019 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
This oral microsomal triglyceride transfer protein (MTP) inhibitor was approved in 2012 in several countries as an adjunct to a low-fat diet and other lipid-lowering drugs with or without LDL apheresis to treat patients with HoFH.
|
30945578 |
2019 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Lomitapide (Juxtapid® in US and Lojuxta® in Europe) is the first developed inhibitor of the microsomal triglyceride transfer protein (MTP) approved as a novel drug for the management of homozygous familial hypercholesterolemia (HoFH).
|
30663562 |
2019 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
GeneticVariation
|
disease |
BEFREE |
We treated two brothers (ages 46 years and 47 years) with HoFH with statins, lipoproteinapheresis (LA) and the microsomal triglyceride transfer protein inhibitor lomitapide.
|
29306853 |
2018 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Lomitapide is a microsomal triglyceride transfer protein inhibitor approved for the treatment of HoFH as an adjunct to LA.
|
27578108 |
2017 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
ApoB antisense and MTP inhibitor are currently approved for patients with homozygous familial hypercholesterolemia.
|
28527325 |
2017 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Here, we explain the steps involved in lipoprotein assembly, summarize the role of MTP in lipoprotein assembly, explore the clinical and molecular basis of HoFH, and review pre-clinical studies and clinical trials with lomitapide and other MTP inhibitors for the treatment of HoFH.
|
27690713 |
2017 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Lomitapide is a microsomal triglyceride transfer protein (MTP) inhibitor approved as an adjunct to other lipid-lowering therapies (LLTs), with or without lipoprotein apheresis (LA), for the treatment of adult HoFH.
|
26228681 |
2016 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
GeneticVariation
|
disease |
BEFREE |
We suggest that in HoFH variants in the MTP gene may impact on the therapeutic response to lomitapide, but this requires further investigation.
|
27170061 |
2016 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Lomitapide is an orally active selective inhibitor of microsomal triglyceride transfer protein approved as adjunctive therapy for homozygous familial hypercholesterolemia (HoFH).
|
27055957 |
2016 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Lomitapide (a microsomal triglyceride transfer protein inhibitor) is an adjunctive treatment for homozygous familial hypercholesterolaemia (HoFH), a rare genetic condition characterised by elevated low-density lipoprotein-cholesterol (LDL-C), and premature, severe, accelerated atherosclerosis.
|
25897792 |
2015 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
AlteredExpression
|
disease |
BEFREE |
Partial MTP inhibition using small molecule inhibitors, such as lomitapide, can effectively lower plasma low-density lipoprotein-cholesterol and apolipoprotein B levels, but is associated with gastrointestinal side effects and hepatic steatosis, whose long-term sequelae remain unclear; lomitapide has accordingly only been approved as a treatment for homozygous familial hypercholesterolemia.
|
25552696 |
2015 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
AlteredExpression
|
disease |
BEFREE |
New therapies such as mipomersen, a second-generation antisense oligonucleotide, microsomal triglyceride transfer protein inhibitors that decrease the synthesis of apolipoprotein B-containing lipoproteins and proprotein convertase subtilisin/kexin type 9 inhibitors hold promise in reducing cholesterol levels in those patients in whom low density lipoprotein cholesterol (LDL-C) reduction is required beyond the use of statins, especially in those with severe heterozygous familial hypercholesterolaemia or homozygous familial hypercholesterolaemia.
|
24870549 |
2014 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
The microsomal triglyceride transfer protein (MTP) inhibitor lomitapide is a licenced adjunct to a low-fat diet and other lipid-lowering medication, with or without low-density lipoprotein apheresis, for the treatment of adults with homozygous familial hypercholesterolaemia (HoFH).
|
25257075 |
2014 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
A microsomal triglyceride transfer protein inhibitor and an antisense oligonucleotide against APOB have recently been approved for use in subjects with clinically diagnosed homozygous familial hypercholesterolemia.
|
24632267 |
2014 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study.
|
23122768 |
2013 |
|
Familial hypercholesterolemia - homozygous
|
0.100 |
Biomarker
|
disease |
BEFREE |
Lomitapide, a systemic MTP inhibitor, significantly reduces LDL-C in homozygous familial hypercholesterolemia (hoFH) when administered concurrently with other lipid-lowering therapies, including apheresis.
|
23594709 |
2013 |