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Orphan designation - USA

  • Substance: Adenoassociated virus vector (AAV) carrying a modified AAV serotype 2 backbone and coding sequence of human thymidine phosphorylase preceded by a human thyroxin-binding globulin promoter
  • ATC code : -
  • EU Number: -
  • Designation date : 04/09/2014
  • Sponsor of orphan designation : COLUMBIA UNIVERSITY MEDICAL CENTER

Designation

Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE).

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