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1 trial matches your criteria

Lysosomal storage disorder

Not yet recruiting

Safety and efficacy of AAV2/8 vector delivering human ARSB gene with prednisone and methylprednisolone in pediatric MPS VI patients – Phase I/II extension

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Methylprednisolone
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