Muscular dystrophy: New challenges and review of the current clinical trials

Current Opinion in Pediatrics

Volumn 25, Issue 6, 2013, Pages 701-707

  Mercuri, Eugenio ^a,b   Muntoni, Francesco ^b  

^a CATHOLIC UNIVERSITY   (Italy)

^b GREAT ORMOND STREET HOSPITAL   (United Kingdom)

Author keywords

muscular dystrophies; natural history; standards of care; therapeutic trials

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ATALUREN; BETA ADRENERGIC RECEPTOR BLOCKING AGENT; COLLAGEN TYPE 6; CORTICOSTEROID; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; LAMININ ALPHA2; MESSENGER RNA PRECURSOR; MITOCHONDRIAL PERMEABILITY TRANSITION PORE; VITAMIN D;

APOPTOSIS; ASSISTED VENTILATION; BECKER MUSCULAR DYSTROPHY; BONE DENSITY; BONE METABOLISM; BREATHING MUSCLE; BULBAR PARALYSIS; CALCIUM INTAKE; CLINICAL TRIAL (TOPIC); CONGESTIVE CARDIOMYOPATHY; COUGHING; DETERIORATION; DISEASE COURSE; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; DYSTROPHINOPATHY; EARLY INTERVENTION; EXERCISE; EXON; EXON SKIPPING; EXPERIMENTAL STUDY; EXTRACELLULAR MATRIX; GOLD STANDARD; HEART CONDUCTION; HEART FUNCTION; HUMAN; HYPERCAPNIA; HYPOXEMIA; IMPLANTABLE CARDIOVERTER DEFIBRILLATOR; INFLAMMATION; LIFE EXPECTANCY; MUSCLE ATROPHY; MUSCLE CONTRACTION; MUSCULAR DYSTROPHY; MUTATIONAL ANALYSIS; MYOTONIC DYSTROPHY; NONHUMAN; NONINVASIVE VENTILATION; NONSENSE MUTATION; OUTCOME ASSESSMENT; PATHOLOGIC FRACTURE; PHENOTYPE; PRACTICE GUIDELINE; PRIORITY JOURNAL; PROTEIN INTAKE; RESPIRATORY FAILURE; REVIEW; STOP CODON; SUDDEN CARDIAC DEATH; TRACHEOSTOMY; VITAMIN SUPPLEMENTATION; WILD TYPE;

ADOLESCENT; ANGIOTENSIN-CONVERTING ENZYME INHIBITORS; ANTI-INFLAMMATORY AGENTS; CHILD; CHILD, PRESCHOOL; CLINICAL TRIALS AS TOPIC; CODON, NONSENSE; FEMALE; GENETIC PREDISPOSITION TO DISEASE; GENETIC THERAPY; HUMANS; INFANT; MALE; MUSCULAR DYSTROPHIES; OLIGORIBONUCLEOTIDES, ANTISENSE; TRANSLATIONAL MEDICAL RESEARCH;

EID: 84888088698     PISSN: 10408703     EISSN: 1531698X     Source Type: Journal    
DOI: 10.1097/MOP.0b013e328365ace5     Document Type: Review

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