Genetics and Emerging Treatments for Duchenne and Becker Muscular Dystrophy

Pediatric Clinics of North America

Volumn 62, Issue 3, 2015, Pages 723-742

  Wein, Nicolas ^a   Alfano, Lindsay ^a,b   Flanigan, Kevin M ^a,c  

^a THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b NATIONWIDE CHILDREN S HOSPITAL   (United States)

^c OHIO STATE UNIVERSITY   (United States)

Author keywords

Becker; DMD gene; Duchenne; Dystrophin; Exon skipping; Gene therapy; Muscular dystrophy; Nonsense suppression

Indexed keywords

ATALUREN; CORTICOSTEROID; CREATINE KINASE; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; DYSTROPHIN; GENTAMICIN; STEROID;

BECKER MUSCULAR DYSTROPHY; BLOOD ANALYSIS; BONE RADIOGRAPHY; BRACE; CARDIOMYOPATHY; CLINICAL FEATURE; CREATINE KINASE BLOOD LEVEL; DRUG EFFICACY; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; EXERCISE; EXON; GENE MUTATION; GENE REPRESSION; GENE TRANSFER; HUMAN; LUNG INSUFFICIENCY; LUNG VENTILATION; MUSCLE BIOPSY; MUSCLE CONTRACTURE; MUSCLE STRENGTH; MUTATIONAL ANALYSIS; NONSENSE MUTATION; OUTCOME ASSESSMENT; PATIENT CARE; POSITIVE END EXPIRATORY PRESSURE; PROTEIN EXPRESSION; REVIEW; SCOLIOSIS; SPINE STABILIZATION; SPLINTING; TREATMENT INDICATION; TREATMENT RESPONSE; GENETICS; INFANT; MALE; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; PRESCHOOL CHILD;

ADRENAL CORTEX HORMONES; CHILD, PRESCHOOL; DYSTROPHIN; HUMANS; INFANT; MALE; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION;

EID: 84929957964     PISSN: 00313955     EISSN: 15578240     Source Type: Journal    
DOI: 10.1016/j.pcl.2015.03.008     Document Type: Review

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