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Volumn 12, Issue 12, 2016, Pages 675-676
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Eteplirsen therapy for Duchenne muscular dystrophy: Skipping to the front of the line
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Author keywords
[No Author keywords available]
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Indexed keywords
ETEPLIRSEN;
GLUCOCORTICOID;
MESSENGER RNA;
OLIGONUCLEOTIDE;
ANTISENSE OLIGONUCLEOTIDE;
MORPHOLINO OLIGONUCLEOTIDE;
AMBULATORY CARE;
BECKER MUSCULAR DYSTROPHY;
CLINICAL EFFECTIVENESS;
CLINICAL OBSERVATION;
DISEASE COURSE;
DOG;
DRUG DEVELOPMENT;
DRUG SAFETY;
DRUG TOLERABILITY;
DUCHENNE MUSCULAR DYSTROPHY;
EUROPE;
EXON SKIPPING;
FOOD AND DRUG ADMINISTRATION;
FUNCTIONAL STATUS;
GENETIC DISORDER;
HEALTH CARE COST;
HEALTH CARE PERSONNEL;
HEALTH ECONOMICS;
HUMAN;
IN VITRO STUDY;
IN VIVO STUDY;
INTERVENTION STUDY;
LOSS OF FUNCTION MUTATION;
METHODOLOGY;
MOTOR PERFORMANCE;
MUSCLE BIOPSY;
NONHUMAN;
NOTE;
OPEN STUDY;
OUTCOME ASSESSMENT;
PHASE 1 CLINICAL TRIAL (TOPIC);
PHASE 2 CLINICAL TRIAL (TOPIC);
PHASE 3 CLINICAL TRIAL (TOPIC);
PREVALENCE;
PROTEIN DEPLETION;
PROTEIN EXPRESSION;
RNA PROCESSING;
SIX MINUTE WALK TEST;
SURVIVAL RATE;
UNITED STATES;
WHEELCHAIR;
ADOLESCENT;
CHILD;
CLINICAL TRIAL (TOPIC);
GENE DELETION;
MALE;
ADOLESCENT;
CHILD;
CLINICAL TRIALS AS TOPIC;
GENE DELETION;
HUMANS;
MALE;
MORPHOLINOS;
MUSCULAR DYSTROPHY, DUCHENNE;
OLIGONUCLEOTIDES, ANTISENSE;
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EID: 84995752459
PISSN: 17594758
EISSN: 17594766
Source Type: Journal
DOI: 10.1038/nrneurol.2016.180 Document Type: Note |
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Times cited : (54)
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References (5)
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