Anthony QUINN,Zachary GOODMAN,Barbara BURTON,Andrew RANKIN,Mark FRIEDMAN,Paresh SONI
申请号:
US16337495
公开号:
US20200038491A1
申请日:
2017.09.28
申请国别(地区):
US
年份:
2020
代理人:
摘要:
The present invention provides methods of reducing liver fibrosis in a human patient with a lysosomal acid lipase (LAL) deficiency comprising administering sebelipase alfa to the patient, wherein the patient has been determined to have at least a one point reduction (e.g., a ≥;1 point reduction or a ≥;2 point reduction) in Ishak fibrosis stage after administration compared to a baseline Ishak fibrosis stage obtained from the patient prior to administration. Also provided are methods of treating a human patient with a lysosomal acid lipase (LAL) deficiency comprising administering sebelipase alfa to the patient, wherein the patient has been determined to have at least a one point reduction (e.g., a ≥;1 point reduction or a ≥;2 point reduction) in Ishak fibrosis stage after administration compared to a baseline Ishak fibrosis stage obtained from the patient prior to administration.