Clinical and Molecular Aspects of Cardiomyopathies Emerging Therapies and Clinical Trials

被引:6
作者
Maurizi, Niccolo [1 ]
Ammirati, Enrico [2 ,3 ]
Coppini, Raffaele [4 ]
Morrone, Amelia [5 ]
Olivotto, Iacopo [1 ]
机构
[1] Careggi Univ Hosp, Cardiomyopathy Unit, Viale Pieraccini 17, I-50132 Florence, Italy
[2] ASST Grande Osped Metropolitano Niguarda, De Gasperis Cardio Ctr, Milan, Italy
[3] ASST Grande Osped Metropolitano Niguarda, Transplant Ctr, Milan, Italy
[4] Univ Florence, Dept NeuroFarBa, Florence, Italy
[5] Meyer Childrens Hosp, Neurosci Dept, Paediat Neurol Unit & Labs, Florence, Italy
关键词
Rare cardiac diseases; New therapies; Precision medicine; Genetics; Cardiomyopathies; Clinical trials; DUCHENNE MUSCULAR-DYSTROPHY; UBIQUITIN-PROTEASOME SYSTEM; TRANSGENIC RABBIT MODEL; MESENCHYMAL STEM-CELLS; LAMIN A/C GENE; HYPERTROPHIC CARDIOMYOPATHY; DILATED CARDIOMYOPATHY; CARDIAC-HYPERTROPHY; HEART-FAILURE; PHARMACOLOGICAL-TREATMENT;
D O I
10.1016/j.hfc.2018.01.001
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Cardiomyopathies are diseases of the myocardium, often genetically determined, associated with heterogeneous phenotypes and clinical manifestations. Despite significant progress in the understanding of these conditions, available treatments mostly target late complications, whereas approaches that promise to interfere with the primary mechanisms and natural history are just beginning to surface. The past decade has witnessed the establishment of large international cardiomyopathy registries, paralleled by advances in cardiac imaging and genetic testing, deeper understanding of the pathophysiology, and growing involvement by the pharmaceutical industry. As a result, the number of molecular interventions under scrutiny is increasing sharply.
引用
收藏
页码:161 / 178
页数:18
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