Indication
Niemann-Pick C Subtype 2
1 clinical trial
7 products
Clinical trial
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGStatus: Recruiting, Estimated PCD: 2025-07-14
Product
Stem Cell TransplantationProduct
IMD Preparative RegimenProduct
OsteopetrosisProduct
CALD SR-AProduct
cALD SR-BProduct
cALD HR-D