Search for an orphan drug

* (*) mandatory field

Other search option(s)

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


Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE).

The documents contained in this web site are presented for information purposes only. The material is in no way intended to replace professional medical care by a qualified specialist and should not be used as a basis for diagnosis or treatment.