Results 61 to 70 of about 1,997 (174)

Resistance to conventional drug therapy and good response to lomitapide allowed the identification of a novel bi-allelic semi-dominant monogenic HoFH: a case report

Current Medical Research and Opinion
Introduction Familial hypercholesterolaemia (FH) is a genetic disorder associated with high cholesterol levels and an increased risk of premature cardiovascular events.
Paolo Fornengo, Simone Mattivi, Elisa Rinaudo, Federica Lepore, Ileana Iemmolo, Valeria Bracciamà, G. B. D. del Prever, A. Faini, Marilena Durazzo, S. Deaglio   +9 more
semanticscholar   +1 more source

Stimulator of interferon genes (STING)‐activating nanomedicines: Translating innate immune modulation into effective therapy for triple‐negative breast cancer

Clinical and Translational Medicine, Volume 16, Issue 1, January 2026.
Graphical representation of STING‐activated nanomedicines is for effective treatment of triple‐negative breast cancer. Abstract Triple‐negative breast cancer (TNBC), marked by profound immunosuppressive complexity, poses a critical challenge in therapy due to the absence of hormone receptors in its phenotype, making it unavailable for conventional ...
Harshita Singhai, Taha Alqahtani, Humood Al Shmrany, Garima Gupta, Umesh Kumar Patil, Amirhossein Sahebkar, Prashant Kesharwani   +6 more
wiley   +1 more source

Genome‐Wide Protein Interaction Analysis in Parasitic Gyrodactylus Flatworms–Fish Hosts System and Drug Target Identification

Advanced Science, Volume 12, Issue 45, December 4, 2025.
Genomic data offer a powerful tool for studying the molecular interactions between parasites and their hosts, but they remain scarce for parasitic monogenean flatworms. This study presents the first high‐quality phased genome assembly for monogeneans (Gyrodactylus kobayashii), and uses it to predict key interacting proteins between monogenean parasite ‐
Dong Zhang, Jie‐Mei Zhao, Chuan‐Yu Xiang, Yi‐Wen Ma, Hong‐Peng Lei, Yu‐Ying Shi, Shun Zhou, Xiaofei Zeng, Jinsong Chen, Fei Liu, Benhe Zeng, Rui Song, Ye Hu, Feng Zhang, Xiang Liu, Wen‐Xiang Li, Gui‐Tang Wang, Ivan Jakovlić   +17 more
wiley   +1 more source

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients with Homozygous Familial Hypercholesterolemia: Study Protocol of the LILITH Study

European Atherosclerosis Journal
Homozygous Familial Hypercholesterolemia (HoFH) is a rare genetic disorder characterized by markedly elevated low-density lipoprotein cholesterol (LDL-C) levels from birth, leading to premature and severe cardiovascular disease.
A. Catapano, Marcello Arca, L. D'Erasmo
semanticscholar   +1 more source

Population Pharmacokinetics and Exposure‐Response Modeling for Evinacumab in Children, Adolescents, and Adults With Homozygous Familial Hypercholesterolemia

CPT: Pharmacometrics &Systems Pharmacology, Volume 14, Issue 11, Page 1823-1834, November 2025.
ABSTRACT Evinacumab, an angiopoietin‐like 3 (ANGPTL3) inhibitor, significantly reduces low‐density lipoprotein cholesterol (LDL‐C), independent of low‐density lipoprotein receptor, in patients with homozygous familial hypercholesterolemia (HoFH). A population pharmacokinetic (PK)/pharmacodynamic (PD) model was previously developed to characterize ...
Sébastien Bihorel, Robert Dingman, Jeanne Mendell, Yuhuan Wang, Poulabi Banerjee, Robert Pordy, John D. Davis, A. Thomas DiCioccio, Lutz Harnisch   +8 more
wiley   +1 more source

Crystal structure of 9-(4-bromobutyl)-9H-fluorene-9-carboxylic acid

Acta Crystallographica Section E, 2014
The title compound, C18H17BrO2, is a key intermediate in the synthesis of lomitapide mesylate, a microsomal triglyceride transfer protein inhibitor. Its asymmetric unit contains two independent molecules with slightly different conformations; the mean ...
Xu-Yang Zhang, Bing-Ni Liu, Ping-Bao Wang, Deng-Ke Liu   +3 more
doaj   +1 more source

Comparison of Model‐Predicted and Observed Evinacumab Pharmacokinetics and Efficacy in Children Aged < 5 Years With Homozygous Familial Hypercholesterolemia

CPT: Pharmacometrics &Systems Pharmacology, Volume 14, Issue 11, Page 1835-1847, November 2025.
ABSTRACT Evinacumab, an angiopoietin‐like 3 inhibitor, significantly reduces low‐density lipoprotein cholesterol (LDL‐C) in patients with homozygous familial hypercholesterolemia (HoFH). Herein, we report pharmacokinetic and efficacy analyses of evinacumab in < 5‐year‐old patients with HoFH.
Sébastien Bihorel, Robert Dingman, Jeanne Mendell, Katy C. Norman, Richard T. George, Xue‐Qiao Zhao, Robert Pordy, Daniel Garcia, Wendy S. Putnam, Geetha Raghuveer, Brian W. McCrindle, Elena Fornari, Ivo Baric, Shubha Srinivasan, Melissa Diamond, Eliot A. Brinton, John D. Davis, A. Thomas DiCioccio, Lutz Harnisch   +18 more
wiley   +1 more source

Why, how and in whom should we measure levels of lipoprotein(a): A review of the latest evidence and clinical implications

Diabetes, Obesity and Metabolism, Volume 27, Issue S8, Page 34-46, September 2025.
Abstract Lipoprotein(a) [Lp(a)] is a genetically determined, causal risk factor for atherosclerotic cardiovascular disease (ASCVD) and calcific aortic valve disease (CAVD). Despite robust evidence from epidemiological and genetic studies, Lp(a) remains underrecognised in clinical practice due to challenges in measurement, lack of guideline familiarity ...
Alexander C. Razavi, Harpreet S. Bhatia, Roger S. Blumenthal, Michael D. Shapiro, Anurag Mehta   +4 more
wiley   +1 more source

Individual analysis of patients with HoFH participating in a phase 3 trial with lomitapide: The Italian cohort [PDF]

, 2016
Background and aims: The efficacy and safety of lomitapide as adjunct treatment for adults with homozygous familial hypercholesterolaemia (HoFH) have been confirmed in a phase 3 trial.
Stefanutti, C., AVERNA, Maurizio, Averna, M., Di Giacomo, S, Sirtori, C.R., Averna, M, CEFALU', Angelo Baldassare, Sirtori, C. R., Di Giacomo, S., VIGNA, Giovanni Battista, Sirtori, C. R, Sirtori, C., STEFANUTTI, Claudia, Vigna, G., Cefalù, A.B., Cefalù, A. B, Cefalù, A. B.   +16 more
core   +1 more source

Use of Potentially Inappropriate Medications Identified by STOPPFrail Among Danish Care Home Residents: A Nationwide Drug Utilisation Study

Basic &Clinical Pharmacology &Toxicology, Volume 137, Issue 2, August 2025.
ABSTRACT Care home residents represent a frail population with limited life expectancy and are often prescribed multiple medications. As therapeutic goals shift in this population, certain treatments may become inappropriate. This study aims to describe potentially inappropriate medication use among Danish care home residents using the Screening Tool ...
Katrine Mose, Carina Lundby, Martin Thomsen Ernst, Jesper Ryg, Anton Pottegård, Lotte Rasmussen   +5 more
wiley   +1 more source