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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