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First received: April 22, 2025

Clinical Trial: A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France

Study Status: COMPLETED

Recruit Status: COMPLETED

Condition: Niemann-Pick Disease

Study Type: INTERVENTIONAL


Official Title: A Long-Term Follow-up Study to Evaluate Safety and Tolerability of Olipudase Alfa in Patients Who Completed the DFI12712 or the LTS13632 Study in France

Brief Summary: This is an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632 Study in France until olipudase alfa reimbursement is granted in France.Study and treatment duration:The period between the patient's completion of Study DFI12712 or LTS13632 and olipudase alfa reimbursement is available in France.In case reimbursement will not be obtained, this study…

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