Results 51 to 60 of about 489 (130)

Yield of Genetic Testing in Pediatric Cardiomyopathies: Implications for Novel Therapeutic Options

Molecular Genetics &Genomic Medicine, Volume 13, Issue 7, July 2025.
Pediatric cardiomyopathies are rare, heterogeneous, and challenging conditions, often with a genetic etiology. We estimated the yield of genetic testing in a pediatric cohort with cardiomyopathies and evaluated the potential candidacy to current or emerging treatments based on genetic results. Over one‐third had a conclusive genetic test, including 25%
Adelaide Ballerini, Francesca Girolami, Alessia Gozzini, Silvia Passantino, Mattia Zampieri, Alberto Marchi, Alessia Tomberli, Giovanni B. Calabri, Gaia Spaziani, Giulio Porcedda, Elena Bennati, Silvia Favilli, Iacopo Olivotto   +12 more
wiley   +1 more source

Efficacy and Safety of Percutaneous Transluminal Septal Myocardial Ablation Treating Hypertrophic Cardiomyopathy: Systematic Review and Meta‐Analysis

International Journal of Clinical Practice, Volume 2025, Issue 1, 2025.
Background: Percutaneous transluminal septal myocardial ablation (PTSMA) is one of the invasive therapeutic measures for the treatment of patients with obstructive hypertrophic cardiomyopathy (HCM). Objective: To systematically evaluate the efficacy and safety of PTSMA in patients with HCM.
Sun Qinyu, Deng Yifan, Yu Jiling, Zhang Zhaoyuan, Cao Gan, Yang Tianxiao, Fang Zhen, Ji Jun, He Shenghu, Zhang Jing, Luigi Sciarra   +10 more
wiley   +1 more source

A Phase 1 Dose-Escalation Study of the Cardiac Myosin Inhibitor Aficamten in Healthy Participants

JACC: Basic to Translational Science, 2022
This phase 1, randomized, double-blind, placebo-controlled study of aficamten (formerly CK-3773274) in healthy adults identified a pharmacologically active range of doses and exposures. At doses that were pharmacologically active (single doses of ≤50 mg or daily dosing of ≤10 mg for 14 or 17 days), aficamten appeared to be safe and well tolerated ...
Malik, Fady I., Robertson, Laura A., Armas, Danielle R., Robbie, Edward P., Osmukhina, Anna, Xu, Donghong, Li, Hanbin, Solomon, Scott D.   +7 more
openaire   +2 more sources

Targeted Therapies in Pediatric and Adult Patients With Hypertrophic Heart Disease: From Molecular Pathophysiology to Personalized Medicine [PDF]

, 2023
Hypertrophic cardiomyopathy is a myocardial disease defined by an increased left ventricular wall thickness not solely explained by abnormal loading conditions.
Altobelli, Ippolita, Bakalakos, Athanasios, De Michele, Gianantonio, Diana, Gaetano, Elliott, Perry M, Falco, Luigi, Limongelli, Giuseppe, Lioncino, Michele, Monda, Emanuele, Palmiero, Giuseppe, Perna, Alessia, Rubino, Marta, Verrillo, Federica   +12 more
core  

Cardiomyopathy: pathogenesis and therapeutic interventions

MedComm, Volume 5, Issue 11, November 2024.
Cardiomyopathy is a group of diseases characterized by structural and functional damage to the myocardium. Many specific gene mutations, environmental factors, and metabolic disorders may cause cardiomyopathy. Traditional therapeutic includes drug and surgery. With the growing comprehension of the molecular mechanisms underlying cardiomyopathy.
Shitong Huang, Jiaxin Li, Qiuying Li, Qiuyu Wang, Xianwu Zhou, Jimei Chen, Xuanhui Chen, Abdelouahab Bellou, Jian Zhuang, Liming Lei   +9 more
wiley   +1 more source

Assessing the Applicability of Cardiac Myosin Inhibitors for Hypertrophic Cardiomyopathy Management in a Large Single Center Cohort

Reviews in Cardiovascular Medicine
Background: Cardiac myosin inhibitors (CMIs), including Mavacamten and Aficamten, have emerged as a groundbreaking treatment for hypertrophic cardiomyopathy (HCM).
Ali Amr, Elham Kayvanpour, Christoph Reich, Jan Koelemen, Shamily Asokan, Norbert Frey, Benjamin Meder, Farbod Sedaghat-Hamedani   +7 more
doaj   +1 more source

From amoeboid myosin to unique targeted medicines for a genetic cardiac disease

Frontiers in Physiology
The importance of fundamental basic research in the quest for much needed clinical treatments is a story that constantly must be retold. Funding of basic science in the USA by the National Institutes of Health and other agencies is provided under the ...
James A. Spudich
doaj   +1 more source

Safety and efficacy of aficamten in patients with non‐obstructive hypertrophic cardiomyopathy: A 36‐week analysis from FOREST‐HCM

European Journal of Heart Failure, Volume 26, Issue 9, Page 1993-1998, September 2024.
Aims The aim of this study was to report safety and efficacy of aficamten in patients with non‐obstructive hypertrophic cardiomyopathy (nHCM) over 36 weeks in the ongoing FOREST‐HCM trial. Methods and results Patients were started on aficamten 5 mg daily, with doses adjusted in 5‐mg increments (5–20 mg) at ≥2‐week intervals according to site‐read left ...
Ahmad Masri, Roberto Barriales‐Villa, Perry Elliott, Michael E. Nassif, Artur Oreziak, Anjali T. Owens, Albree Tower‐Rader, Stephen B. Heitner, Stuart Kupfer, Fady I. Malik, Chiara Melloni, Lisa Meng, Jenny Wei, Sara Saberi, on behalf of the FOREST‐HCM Investigators   +14 more
wiley   +1 more source

A systematic review of present and future pharmaco‐structural therapies for hypertrophic cardiomyopathy

Clinical Cardiology, Volume 47, Issue 1, January 2024.
Hypertrophic cardiomyopathy (HCM) is an increasingly recognized genetic condition that is accompanied by a heterogeneous phenotype. The presence of outflow tract obstruction carries an increased risk for the development of heart failure, arrhythmias, and sudden cardiac death.
Mariem A. Sawan, Sindhu Prabakaran, Melroy D'Souza, Omid Behbahani‐Nejad, Matthew E. Gold, Byron Robinson Williams, Ozlem Bilen   +6 more
wiley   +1 more source

What's new in heart failure? October 2024 [PDF]

No abstract ...
Aimo, Alberto, Codina, Pau, Lee, Matthew M. Y., Tomasoni, Daniela   +3 more
core   +2 more sources