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Orphan designation - Europe

  • Substance: Antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA
  • ATC code : -
  • EU Number: EU/3/16/1641
  • Designation date : 28/04/2016
  • Sponsor of orphan designation : PROQR THERAPEUTICS B.V.
  • Summary of Opinion


Treatment of Leber's congenital amaurosis

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