Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Status:
Terminated
Trial end date:
2018-10-30
Target enrollment:
Participant gender:
Summary
This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly
progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received
once-weekly infusions of sebelipase alfa for up to 3 years.