FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

Nucleic Acid Therapeutics

Volumn 27, Issue 2, 2017, Pages 67-69

  Aartsma Rus, Annemieke ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

antisense oligonucleotide; eteplirsen; nusinersen; spinal muscular atrophy; therapy

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ETEPLIRSEN; MESSENGER RNA; MORPHOLINO OLIGONUCLEOTIDE; NUSINERSEN; OLIGONUCLEOTIDE PHOSPHOROTHIOATE; PLACEBO; SURVIVAL MOTOR NEURON PROTEIN; OLIGONUCLEOTIDE; SMN1 PROTEIN, HUMAN; SMN2 PROTEIN, HUMAN; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN 2;

ANTISENSE THERAPY; BLOOD BRAIN BARRIER; CENTRAL NERVOUS SYSTEM; CLINICAL EVALUATION; DRUG APPROVAL; HEALTH INSURANCE; HISTORY; HUMAN; INTRON; MOLECULAR RECOGNITION; MOTONEURON; MOTOR PERFORMANCE; MUSCLE FUNCTION; NEUROMUSCULAR DISEASE; NONHUMAN; OPEN STUDY; PHASE 1 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; REVIEW; SPINAL MUSCULAR ATROPHY; THERAPY EFFECT; UNITED STATES; ADOLESCENT; ADULT; ALTERNATIVE RNA SPLICING; ANIMAL; ANIMAL MODEL; CHILD; DRUG EFFECT; ECONOMICS; FOOD AND DRUG ADMINISTRATION; GENETICS; INFANT; PRESCHOOL CHILD;

ADOLESCENT; ADULT; ALTERNATIVE SPLICING; ANIMALS; CHILD; CHILD, PRESCHOOL; DRUG APPROVAL; HUMANS; INFANT; MODELS, ANIMAL; MORPHOLINOS; MUSCULAR ATROPHY, SPINAL; OLIGONUCLEOTIDES; OLIGONUCLEOTIDES, ANTISENSE; SURVIVAL OF MOTOR NEURON 1 PROTEIN; SURVIVAL OF MOTOR NEURON 2 PROTEIN; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 85016512565     PISSN: 21593337     EISSN: 21593345     Source Type: Journal    
DOI: 10.1089/nat.2017.0665     Document Type: Review

References (16)

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