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Is there hope for spinal muscular atrophy synthetic pharmacotherapy?

Research output: Contribution to journalEditorial

2 Scopus citations
Original languageEnglish
Pages (from-to)1049-1052
Number of pages4
JournalExpert Opinion on Pharmacotherapy
Volume20
Issue number9
DOIs
StatePublished - Jun 13 2019

Keywords

  • Antisense oligonucleotides (ASO)
  • nusinersen
  • spinal cord
  • spinal muscular atrophy
  • splicing correction therapy
  • survival of motor neuron (SMN2)

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