Hypertrophic cardiomyopathy: the future of treatment
Hypertrophic cardiomyopathy (HCM) is a heterogeneous genetic disorder most often caused by sarcomeric mutations resulting in left ventricular hypertrophy, fibrosis, hypercontractility, and reduced compliance. It is the most common inherited monogenic cardiac condition, affecting 0.2% of the populati...
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| Veröffentlicht in: | European journal of heart failure 2020-02, Vol.22 (2), p.228-240 |
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| container_title | European journal of heart failure |
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| creator | Tuohy, C. Vaughan Kaul, Sanjiv Song, Howard K. Nazer, Babak Heitner, Stephen B. |
| description | Hypertrophic cardiomyopathy (HCM) is a heterogeneous genetic disorder most often caused by sarcomeric mutations resulting in left ventricular hypertrophy, fibrosis, hypercontractility, and reduced compliance. It is the most common inherited monogenic cardiac condition, affecting 0.2% of the population. Whereas currently available therapies for HCM have been effective in reducing morbidity, there remain important unmet needs in the treatment of both the obstructive and non‐obstructive phenotypes. Novel pharmacotherapies directly target the molecular underpinnings of HCM, while innovative procedural techniques may soon offer minimally‐invasive alternatives to current septal reduction therapy. With the advent of embryonic gene editing, there now exists the potential to correct underlying genetic mutations that may result in disease. This article details the recent developments in the treatment of HCM including pharmacotherapy, septal reduction procedures, mitral valve manipulation, and gene‐based therapies. |
| doi_str_mv | 10.1002/ejhf.1715 |
| format | Article |
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Vaughan ; Kaul, Sanjiv ; Song, Howard K. ; Nazer, Babak ; Heitner, Stephen B.</creator><creatorcontrib>Tuohy, C. Vaughan ; Kaul, Sanjiv ; Song, Howard K. ; Nazer, Babak ; Heitner, Stephen B.</creatorcontrib><description>Hypertrophic cardiomyopathy (HCM) is a heterogeneous genetic disorder most often caused by sarcomeric mutations resulting in left ventricular hypertrophy, fibrosis, hypercontractility, and reduced compliance. It is the most common inherited monogenic cardiac condition, affecting 0.2% of the population. Whereas currently available therapies for HCM have been effective in reducing morbidity, there remain important unmet needs in the treatment of both the obstructive and non‐obstructive phenotypes. Novel pharmacotherapies directly target the molecular underpinnings of HCM, while innovative procedural techniques may soon offer minimally‐invasive alternatives to current septal reduction therapy. With the advent of embryonic gene editing, there now exists the potential to correct underlying genetic mutations that may result in disease. This article details the recent developments in the treatment of HCM including pharmacotherapy, septal reduction procedures, mitral valve manipulation, and gene‐based therapies.</description><identifier>ISSN: 1388-9842</identifier><identifier>EISSN: 1879-0844</identifier><identifier>DOI: 10.1002/ejhf.1715</identifier><identifier>PMID: 31919938</identifier><language>eng</language><publisher>Oxford, UK: John Wiley & Sons, Ltd</publisher><subject>Cardiac Surgical Procedures ; Cardiomyopathy, Hypertrophic - genetics ; Cardiomyopathy, Hypertrophic - therapy ; CRISPR/Cas9 ; Genome editing ; Heart Failure ; High‐intensity focused ultrasound ; Humans ; Hypertrophic cardiomyopathy ; Mavacamten ; MitraClip ; Mitral Valve ; Myectomy ; Papillary muscle ; Percutaneous mitral valve repair ; Radiofrequency ablation ; Treatment Outcome</subject><ispartof>European journal of heart failure, 2020-02, Vol.22 (2), p.228-240</ispartof><rights>2020 The Authors. © 2020 European Society of Cardiology</rights><rights>2020 The Authors. 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Novel pharmacotherapies directly target the molecular underpinnings of HCM, while innovative procedural techniques may soon offer minimally‐invasive alternatives to current septal reduction therapy. With the advent of embryonic gene editing, there now exists the potential to correct underlying genetic mutations that may result in disease. This article details the recent developments in the treatment of HCM including pharmacotherapy, septal reduction procedures, mitral valve manipulation, and gene‐based therapies.</description><subject>Cardiac Surgical Procedures</subject><subject>Cardiomyopathy, Hypertrophic - genetics</subject><subject>Cardiomyopathy, Hypertrophic - therapy</subject><subject>CRISPR/Cas9</subject><subject>Genome editing</subject><subject>Heart Failure</subject><subject>High‐intensity focused ultrasound</subject><subject>Humans</subject><subject>Hypertrophic cardiomyopathy</subject><subject>Mavacamten</subject><subject>MitraClip</subject><subject>Mitral Valve</subject><subject>Myectomy</subject><subject>Papillary muscle</subject><subject>Percutaneous mitral valve repair</subject><subject>Radiofrequency ablation</subject><subject>Treatment Outcome</subject><issn>1388-9842</issn><issn>1879-0844</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp10L1OwzAUhmELgWgpDNwAyghDWv8mNhuqWgqqxAKz5TgnSqqkCbYjlLsnpYWN6Zzh0Te8CN0SPCcY0wXsymJOUiLO0JTIVMVYcn4-_kzKWElOJ-jK-x3GJB35JZowoohSTE4R3wwduODarqxsZI3Lq7YZ2s6EcniMQglR0YfeQdQWUXBgQgP7cI0uClN7uDndGfpYr96Xm3j79vyyfNrGltNExIoklFGaUW4l5qAsGJ5SleQCZ8yqjBdc5SnNTJIrKwiIzKgUS8oYVQIA2AzdH3c713724INuKm-hrs0e2t7rUSZUJDLhI304Uuta7x0UunNVY9ygCdaHSPoQSR8ijfbuNNtnDeR_8rfKCBZH8FXVMPy_pFevm_XP5Df7vHBh</recordid><startdate>202002</startdate><enddate>202002</enddate><creator>Tuohy, C. 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Vaughan</creatorcontrib><creatorcontrib>Kaul, Sanjiv</creatorcontrib><creatorcontrib>Song, Howard K.</creatorcontrib><creatorcontrib>Nazer, Babak</creatorcontrib><creatorcontrib>Heitner, Stephen B.</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>European journal of heart failure</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Tuohy, C. Vaughan</au><au>Kaul, Sanjiv</au><au>Song, Howard K.</au><au>Nazer, Babak</au><au>Heitner, Stephen B.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Hypertrophic cardiomyopathy: the future of treatment</atitle><jtitle>European journal of heart failure</jtitle><addtitle>Eur J Heart Fail</addtitle><date>2020-02</date><risdate>2020</risdate><volume>22</volume><issue>2</issue><spage>228</spage><epage>240</epage><pages>228-240</pages><issn>1388-9842</issn><eissn>1879-0844</eissn><abstract>Hypertrophic cardiomyopathy (HCM) is a heterogeneous genetic disorder most often caused by sarcomeric mutations resulting in left ventricular hypertrophy, fibrosis, hypercontractility, and reduced compliance. It is the most common inherited monogenic cardiac condition, affecting 0.2% of the population. Whereas currently available therapies for HCM have been effective in reducing morbidity, there remain important unmet needs in the treatment of both the obstructive and non‐obstructive phenotypes. Novel pharmacotherapies directly target the molecular underpinnings of HCM, while innovative procedural techniques may soon offer minimally‐invasive alternatives to current septal reduction therapy. With the advent of embryonic gene editing, there now exists the potential to correct underlying genetic mutations that may result in disease. This article details the recent developments in the treatment of HCM including pharmacotherapy, septal reduction procedures, mitral valve manipulation, and gene‐based therapies.</abstract><cop>Oxford, UK</cop><pub>John Wiley & Sons, Ltd</pub><pmid>31919938</pmid><doi>10.1002/ejhf.1715</doi><tpages>13</tpages><oa>free_for_read</oa></addata></record> |
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| ispartof | European journal of heart failure, 2020-02, Vol.22 (2), p.228-240 |
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| source | Wiley Online Library - AutoHoldings Journals; MEDLINE; Wiley Online Library; EZB Electronic Journals Library |
| subjects | Cardiac Surgical Procedures Cardiomyopathy, Hypertrophic - genetics Cardiomyopathy, Hypertrophic - therapy CRISPR/Cas9 Genome editing Heart Failure High‐intensity focused ultrasound Humans Hypertrophic cardiomyopathy Mavacamten MitraClip Mitral Valve Myectomy Papillary muscle Percutaneous mitral valve repair Radiofrequency ablation Treatment Outcome |
| title | Hypertrophic cardiomyopathy: the future of treatment |
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