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

Aartsma-Rus, Annemieke

FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides. [electronic resource] - Nucleic acid therapeutics 04 2017 - 67-69 p. digital

Publication Type: Journal Article

2159-3345

10.1089/nat.2017.0665 doi


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