Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

Nature Neuroscience

Volumn 20, Issue 4, 2017, Pages 497-499

  Corey, David R ^a  

^a UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; NUSINERSEN; OLIGONUCLEOTIDE;

ARTICLE; AUTOPSY; DRUG DEVELOPMENT; DRUG EFFICACY; DRUG SAFETY; DRUG STRUCTURE; EXON; FOLLOW UP; GENE; GENE MUTATION; GENETIC ASSOCIATION; HUMAN; INTRON; LUMBAR PUNCTURE; MOLECULAR WEIGHT; MOUSE; NONHUMAN; OPEN STUDY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); RISK BENEFIT ANALYSIS; RNA SPLICING; SINGLE DRUG DOSE; SMN1 GENE; SMN2 GENE; SPINAL MUSCULAR ATROPHY; TREATMENT RESPONSE; ANIMAL; PATHOPHYSIOLOGY; PROCEDURES; TRENDS;

ANIMALS; DRUG DISCOVERY; HUMANS; MUSCULAR ATROPHY, SPINAL; OLIGONUCLEOTIDES; OLIGONUCLEOTIDES, ANTISENSE;

EID: 85012300517     PISSN: 10976256     EISSN: 15461726     Source Type: Journal    
DOI: 10.1038/nn.4508     Document Type: Article

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