Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Inclusion Criteria

• •1. Participant was \>8 months of age at the time of dosing.
• •2. Confirmation of LAL-D diagnosis as determined by the central laboratory or, for participants with prior hematopoietic stem cell transplant or liver transplant, historical enzyme activity or molecular genetic testing confirming a diagnosis of LAL-D.
• •3. Participants \>8 months but \<4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
• •* Dyslipidemia
• •* Elevated transaminases
• •* Impaired growth
• •* Suspected malabsorption
• •* Other clinical manifestation of LAL-D
• •4. Participants ≥4 years of age at Screening had at least 1 of the following documented clinical manifestations of LAL-D:
• •* Evidence of advanced liver disease
• •* Histologically confirmed disease recurrence in participants with past liver or hematopoietic transplant
• •* Persistent dyslipidemia
• •* Suspected malabsorption
• •* Other clinical manifestation of LAL-D