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Orphan designation - Europe

  • Substance: Adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes
  • ATC code : -
  • EU Number: EU/3/16/1796
  • Designation date : 12/12/2016
  • Sponsor of orphan designation : FONDAZIONE TELETHON
  • Summary of Opinion


Treatment of retinitis pigmentosa.

Diseases list

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