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 will end 5 years after starting. Visit frequency: every 2 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion
Investigational Site Number : 2500002
Bron, France
Investigational Site Number : 2500001
Paris, France
Number of adverse events (AEs) / serious adverse events (SAEs)
Time frame: Baseline to approximately 5 years
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