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EBGene trial: patient preselection outcomes for the European GENEGRAFT ex vivo phase I/II gene therapy trial for recessive dystrophic epidermolysis bullosa.

S GaucherSu M LwinMatthias TiteuxA Abdul-WahabNathalie PirononAraksya IzmiryanSnaigune MiskinyteClarisse GanierS DuchateletJemima E MellerioEmmanuelle BourratJohn A McGrathAlain Hovnanian
Published in: The British journal of dermatology (2019)
Keyphrases
  • gene therapy
  • phase iii
  • study protocol
  • phase ii
  • clinical trial
  • open label
  • intellectual disability
  • randomized controlled trial
  • type diabetes
  • autism spectrum disorder
  • muscular dystrophy
  • double blind