Acid Sphingomyelinase Deficiency (ASMD) Clinical Trial
Official title:
Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France
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
Approximate duration of enrollment: 18 months Total study duration: approximately 3 years This is a national, multicenter observational retrospective and prospective cohort data collection study. Retrospective is defined as collection of data from all patients, including deceased patients, who were already on early access olipudase alfa in France before the start of this study. ;