Novel myosin-based therapies for congenital cardiac and skeletal myopathies

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myopathy 14 rheumatologydiseases

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Select Disease Character Offset Disease Term Instance
myopathy 1197 myofilament level grandly contributing to a wide range of clinical phenotypes such as hypertrophic cardio myopathy (HCM), dilated cardiomyopathy (DCM), restrictive cardiomyopathy (RCM), nemaline myopathy or Laing early
myopathy 1227 contributing to a wide range of clinical phenotypes such as hypertrophic cardiomyopathy (HCM), dilated cardio myopathy (DCM), restrictive cardiomyopathy (RCM), nemaline myopathy or Laing early onset distal myopathy.[2][3]
myopathy 1261 phenotypes such as hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM), restrictive cardio myopathy (RCM), nemaline myopathy or Laing early onset distal myopathy.[2][3] No treatment exists for these sarcomeric
myopathy 1286 hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM), restrictive cardiomyopathy (RCM), nemaline myopathy or Laing early onset distal myopathy.[2][3] No treatment exists for these sarcomeric diseases. The current
myopathy 1323 cardiomyopathy (DCM), restrictive cardiomyopathy (RCM), nemaline myopathy or Laing early onset distal myopathy .[2][3] No treatment exists for these sarcomeric diseases. The current guidelines state that therapeutic
myopathy 2471 vigorous systolic function.[6] They can also induce DCM, RCM and left ventricular non-compaction cardio myopathy , which are also characterised by enlarged left ventricles but with various effects on systolic function.[7]
myopathy 3044 and breathing.[10][11] The following is a non-exhaustive list of the most common diseases: nemaline myopathy ,[11] Laing early onset distal myopathy,[12] actin myopathy,[13] intranuclear rod myopathy,[13] rod-core
myopathy 3083 a non-exhaustive list of the most common diseases: nemaline myopathy,[11] Laing early onset distal myopathy ,[12] actin myopathy,[13] intranuclear rod myopathy,[13] rod-core disease,[13] congenital fibre-type
myopathy 3103 list of the most common diseases: nemaline myopathy,[11] Laing early onset distal myopathy,[12] actin myopathy ,[13] intranuclear rod myopathy,[13] rod-core disease,[13] congenital fibre-type disproportion,[13] hyaline
myopathy 3134 nemaline myopathy,[11] Laing early onset distal myopathy,[12] actin myopathy,[13] intranuclear rod myopathy ,[13] rod-core disease,[13] congenital fibre-type disproportion,[13] hyaline body myopathy,[12] myosin
myopathy 3224 intranuclear rod myopathy,[13] rod-core disease,[13] congenital fibre-type disproportion,[13] hyaline body myopathy ,[12] myosin storage myopathy,[12] inclusion body myopathy[12] and myofibrillar myopathy.[14] Interestingly,
myopathy 3253 rod-core disease,[13] congenital fibre-type disproportion,[13] hyaline body myopathy,[12] myosin storage myopathy ,[12] inclusion body myopathy[12] and myofibrillar myopathy.[14] Interestingly, a few of the above mutations,
myopathy 3282 fibre-type disproportion,[13] hyaline body myopathy,[12] myosin storage myopathy,[12] inclusion body myopathy [12] and myofibrillar myopathy.[14] Interestingly, a few of the above mutations, especially in the MYH7
myopathy 3312 hyaline body myopathy,[12] myosin storage myopathy,[12] inclusion body myopathy[12] and myofibrillar myopathy .[14] Interestingly, a few of the above mutations, especially in the MYH7 gene, are associated with cardiac

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