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Circulation, ISSN 0009-7322, 08/2017, Volume 136, Issue 13, pp. e200 - e231
For many neuromuscular diseases (NMDs), cardiac disease represents a major cause of morbidity and mortality. The management of cardiac disease in NMDs is made... 
Neuromuscular disease | Pediatrics | Cardiomyopathy | Muscular dystrophy | AHA Scientific Statements | CARDIAC & CARDIOVASCULAR SYSTEMS | CARDIOVASCULAR MAGNETIC-RESONANCE | MECHANICAL CIRCULATORY SUPPORT | neuromuscular disease | CONVERTING ENZYME-INHIBITORS | LUNG TRANSPLANTATION GUIDELINES | LEFT-VENTRICULAR NONCOMPACTION | muscular dystrophy | pediatrics | CHEYNE-STOKES RESPIRATION | LAMIN A/C GENE | PERIPHERAL VASCULAR DISEASE | DUCHENNE MUSCULAR-DYSTROPHY | MYOCARDIAL DELAYED ENHANCEMENT | cardiomyopathy | CARDIOSELECTIVE BETA-BLOCKERS | Myotonic Dystrophy - metabolism | Barth Syndrome - metabolism | Friedreich Ataxia - pathology | Myotonic Dystrophy - diagnosis | United States | Humans | Myopathies, Structural, Congenital - genetics | Neuromuscular Diseases - pathology | Barth Syndrome - pathology | Myotonic Dystrophy - pathology | Muscular Dystrophy, Emery-Dreifuss - diagnosis | Cardiomyopathies - diagnosis | Neuromuscular Diseases - diagnosis | Muscular Dystrophies, Limb-Girdle - pathology | Friedreich Ataxia - metabolism | Muscular Dystrophies, Limb-Girdle - diagnosis | Muscular Diseases - metabolism | Risk Factors | Friedreich Ataxia - diagnosis | Muscular Dystrophy, Emery-Dreifuss - pathology | Cardiomyopathies - pathology | Muscular Dystrophy, Duchenne - pathology | Muscular Diseases - pathology | Barth Syndrome - diagnosis | American Heart Association | Muscular Diseases - diagnosis | Muscular Dystrophy, Emery-Dreifuss - metabolism | Muscular Dystrophies, Limb-Girdle - metabolism | Barth Syndrome - genetics | Neuromuscular Diseases - complications | Myopathies, Structural, Congenital - pathology | Cardiomyopathies - complications | Myopathies, Structural, Congenital - diagnosis | Muscular Dystrophy, Duchenne - metabolism | Myopathies, Structural, Congenital - metabolism | Muscular Dystrophy, Duchenne - diagnosis | Index Medicus | Abridged Index Medicus
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 12/2012, Volume 122, Issue 12, pp. 4461 - 4472
Journal Article
Nature, ISSN 0028-0836, 08/2012, Volume 488, Issue 7409, pp. 111 - 115
Antisense oligonucleotides (ASOs) hold promise for gene-specific knockdown in diseases that involve RNA or protein gain-of-function effects. In the hereditary... 
CTG REPEAT | 3' END | PROTEIN | MESSENGER-RNA | MULTIDISCIPLINARY SCIENCES | GENE-EXPRESSION | TOXIC RNA | MODIFIED ANTISENSE OLIGONUCLEOTIDE | PHOSPHOROTHIOATE OLIGONUCLEOTIDES | MOUSE MODELS | TRINUCLEOTIDE REPEAT | Humans | Muscle, Skeletal - metabolism | RNA, Messenger - metabolism | RNA, Untranslated - genetics | Gene Knockdown Techniques | RNA - genetics | Myotonic Dystrophy - pathology | Base Sequence | Muscle, Skeletal - drug effects | Oligonucleotides, Antisense - therapeutic use | RNA, Messenger - antagonists & inhibitors | Mice, Inbred mdx | Myotonic Dystrophy - genetics | Disease Models, Animal | RNA - metabolism | Oligonucleotides, Antisense - pharmacology | Myotonin-Protein Kinase | RNA - antagonists & inhibitors | Mice, Inbred C57BL | RNA, Messenger - genetics | Gene Silencing | Protein-Serine-Threonine Kinases - genetics | Mice, Transgenic | Myotonic Dystrophy - therapy | Transcriptome - drug effects | Transcriptome - genetics | Myotonic Dystrophy - physiopathology | Animals | Oligonucleotides, Antisense - genetics | Cell Nucleus - genetics | RNA, Long Noncoding | Alleles | Trinucleotide Repeat Expansion - genetics | Mice | Mice, Inbred BALB C | Cell Nucleus - drug effects | Ribonuclease H - metabolism | Myotonic dystrophy | RNA | Oligonucleotides | Physiological aspects | Development and progression | Genetic aspects | Health aspects | Index Medicus
Journal Article
Journal Article
Journal Article
Science, ISSN 0036-8075, 12/2003, Volume 302, Issue 5652, pp. 1978 - 1980
The neuromuscular disease myotonic dystrophy (DM) is caused by microsatellite repeat expansions at two different genomic loci. Mutant DM transcripts are... 
Proteins | Cataracts | Splicing | Reverse transcriptase polymerase chain reaction | Myotonic dystrophy | Complementary DNA | Exons | Myotonia | Genes | Hypoxia | Reports | SKELETAL-MUSCLE | CELLS | MULTIDISCIPLINARY SCIENCES | IN-VIVO | EXPANSION | NUCLEAR FOCI | PROTEINS | DROSOPHILA | EXPANDED-REPEAT TRANSCRIPTS | PRE-MESSENGER-RNA | TRINUCLEOTIDE REPEAT | RNA-Binding Proteins - genetics | Alternative Splicing | Cataract - pathology | Chloride Channels - genetics | Troponin T - metabolism | Myotonic Dystrophy - pathology | RNA Splicing | Cell Nucleus - metabolism | Myocardium - metabolism | Myotonic Dystrophy - genetics | Electromyography | DNA-Binding Proteins | Disease Models, Animal | Troponin T - genetics | Gene Targeting | RNA-Binding Proteins - physiology | Cataract - etiology | Introns | Mice, Inbred C57BL | CELF1 Protein | Muscle Relaxation | Reverse Transcriptase Polymerase Chain Reaction | Trinucleotide Repeat Expansion | Mice, Knockout | Chloride Channels - metabolism | Myotonic Dystrophy - physiopathology | Animals | Muscle, Skeletal - physiopathology | Protein Isoforms | Muscle Fibers, Skeletal - pathology | Mice | Muscle, Skeletal - pathology | RNA-Binding Proteins - metabolism | Usage | Genetic aspects | Binding proteins | Properties | Sequestration (Chemistry) | Biochemistry | Muscular system | Ribonucleic acid--RNA | Muscular dystrophy | myotonic dystrophy | neuromuscular diseases | Mbnl1 gene | Index Medicus
Journal Article
EMBO Molecular Medicine, ISSN 1757-4676, 12/2013, Volume 5, Issue 12, pp. 1887 - 1900
Journal Article