Results 31 to 40 of about 461 (138)

Precision medicine in complex diseases—Molecular subgrouping for improved prediction and treatment stratification

open access: yesJournal of Internal Medicine, Volume 294, Issue 4, Page 378-396, October 2023., 2023
Abstract Complex diseases are caused by a combination of genetic, lifestyle, and environmental factors and comprise common noncommunicable diseases, including allergies, cardiovascular disease, and psychiatric and metabolic disorders. More than 25% of Europeans suffer from a complex disease, and together these diseases account for 70% of all deaths ...
Åsa Johansson   +9 more
wiley   +1 more source

A pathogenic nonsense mutation (c.1522C>T) of the MYBPC3 gene is implicated with hypertrophic cardiomyopathy

open access: yesESC Heart Failure, Volume 10, Issue 4, Page 2711-2717, August 2023., 2023
Abstract Hypertrophic cardiomyopathy (HCM), a genetically and clinically heterogeneous cardiomyopathy, is commonly caused by mutations in the MYBPC3 gene or other various sarcomeric genes. HCM patients carrying sarcomeric gene mutations may experience an asymptomatic period at early stage but still possess an escalating risk of developing adverse ...
Erru Ni   +3 more
wiley   +1 more source

Is mavacamten superior to aficamten for hypertrophic cardiomyopathy? A frequentist network meta-analysis [PDF]

open access: yesExploration of Cardiology
Background: Myosin inhibitors have been shown to improve exercise capacity and symptoms, as well as reduce the left ventricular outflow tract (LVOT) gradient. This study explores the efficacy of mavacamten versus aficamten in hypertrophic cardiomyopathy (
Ayesha Aman   +9 more
doaj   +1 more source

Effect of Hepatic Impairment or Renal Impairment on the Pharmacokinetics of Aficamten. [PDF]

open access: yesClin Pharmacokinet
Aficamten, a small-molecule, selective cardiac myosin inhibitor, is under development for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Aficamten is primarily eliminated by hepatic metabolism with renal excretion playing a minor role.
Xu D   +8 more
europepmc   +3 more sources

Efficacy of aficamten in patients with obstructive hypertrophic cardiomyopathy and mild symptoms: results from the SEQUOIA-HCM trial. [PDF]

open access: yesEur Heart J
Background and Aims: Patients with obstructive hypertrophic cardiomyopathy (oHCM) treated with aficamten in SEQUOIA-HCM (NCT05186818) demonstrated marked improvement in symptoms and functional capacity.
Maron MS   +17 more
europepmc   +2 more sources

Impact of aficamten on disease and symptom burden in obstructive hypertrophic cardiomyopathy results from SEQUOIA-HCM [PDF]

open access: yes
Background: Aficamten is a cardiac myosin inhibitor that mitigates left ventricular outflow gradients in obstructive hypertrophic cardiomyopathy (oHCM).
van Sinttruije, Marion   +51 more
core   +2 more sources

Hypertrophic cardiomyopathy: Mutations to mechanisms to therapies

open access: yesFrontiers in Physiology, 2022
Hypertrophic cardiomyopathy (HCM) affects more than 1 in 500 people in the general population with an extensive burden of morbidity in the form of arrhythmia, heart failure, and sudden death.
Masataka Kawana   +3 more
doaj   +1 more source

Left ventricular outflow tract obstruction in hypertrophic cardiomyopathy and the impact of mavacamten

open access: yesTherapeutic Advances in Chronic Disease, 2022
Hypertrophic cardiomyopathy (HCM) is a common genetic disorder characterised by unexplained left ventricular hypertrophy. Left ventricular outflow tract obstruction is an integral component of the disease, often resulting in significant symptoms, but ...
Susan Mathai, Lynne Williams
doaj   +1 more source

Table1_Safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of aficamten in healthy Chinese participants: a randomized, double-blind, placebo-controlled, phase 1 study.DOCX

open access: yes, 2023
Objectives: Aficamten is a selective, small-molecule allosteric inhibitor of cardiac sarcomere being developed as a chronic oral treatment for patients with symptomatic obstructive hypertrophic cardiomyopathy.
Aijing Liu (662185)   +35 more
core   +1 more source

A New Chapter in the Management of Hypertrophic Cardiomyopathy: Cardiac Myosin Inhibitors [PDF]

open access: yes, 2023
In the recent years, there has been a significant breakthrough in the treatment of hypertrophic cardiomyopathy. New precision molecules have been developed and successfully applied in clinical trials. A new class of orally available allosteric inhibitors
Sepp, Róbert   +5 more
core   +2 more sources

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