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Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
标题(英文)
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
条件
Gaucher Disease, Type 2
干预
Biological: PR001|Drug: Methylprednisolone|Drug: Sirolimus|Drug: Prednisone