Not yet recruiting
Lysosomal storage disorder+1
Not yet recruitingSafety 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
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:
Italy
Italy- Participants:0–17 years
- Substances:Methylprednisolone
- Sponsor:Fondazione Telethon Ets