Primary Objective: To describe the lung, spleen and liver outcomes of olipudase alfa Secondary Objectives: * To describe the patient's characteristics * To describe conditions of olipudase alfa use * To describe safety data related to the use of olipudase alfa * To describe complementary effectiveness outcomes parameters
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Change in pulmonary function diffusion capacity of lung for carbon monoxide (DLco)
Timeframe: From baseline to 24 months
Change in spleen size
Timeframe: From baseline to 24 months
Change in liver size
Timeframe: From baseline to 24 months