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Osteopetrosis

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1 Clinical trial

Osteopetrosis

Recruiting

Study of gene-modified stem cells (FT024) in children with TCIRG1-related osteopetrosis using treosulfan, thiotepa, and plerixafor for treatment

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Treosulfan
  • Sponsor:Fondazione Telethon Ets
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