ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy

Translational Neuroscience

Volumn 8, Issue 1, 2017, Pages 1-6

  Ottesen, Eric W ^a  

^a IOWA STATE UNIVERSITY   (United States)

Author keywords

Antisense oligonucleotides; IONIS SMNRX; ISIS SMNRX; ISS N1; Nusinersen; SMN; Spinal muscular atrophy; Spinraza; Splicing regulation; Survival motor neuron protein

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; NUSINERSEN; SURVIVAL MOTOR NEURON PROTEIN 1;

CLINICAL PROTOCOL; DRUG APPROVAL; EXON SKIPPING; FOOD AND DRUG ADMINISTRATION; GENE DELETION; HUMAN; PRIORITY JOURNAL; REVIEW; SPINAL MUSCULAR ATROPHY;

EID: 85015643038     PISSN: 20813856     EISSN: 20816936     Source Type: Journal    
DOI: 10.1515/tnsci-2017-0001     Document Type: Review

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