FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides. [electronic resource]
Producer: 20180801Description: 67-69 p. digitalISSN:- 2159-3345
- Adolescent
- Adult
- Alternative Splicing -- drug effects
- Animals
- Child
- Child, Preschool
- Drug Approval
- Humans
- Infant
- Models, Animal
- Morpholinos -- therapeutic use
- Muscular Atrophy, Spinal -- drug therapy
- Oligonucleotides -- economics
- Oligonucleotides, Antisense -- economics
- Survival of Motor Neuron 1 Protein -- genetics
- Survival of Motor Neuron 2 Protein -- genetics
- United States
- United States Food and Drug Administration
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Publication Type: Journal Article
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