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SBC-102 long-term extension study in children with lysosomal acid lipase deficiency
2011-002880-42Phase 2Terminated
- Sponsor
- Synageva BioPharma
- Start
- Sep 2011
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of sebelipase alfa in lysosomal acid lipase deficiency (LALD) and Wolman disease, sponsored by Synageva BioPharma. Primary endpoint: Proportion of subjects surviving to 12 months of age. Started Sep 2011; status: terminated.
02
Drugs
sebelipase alfa
03
Conditions
lysosomal acid lipase deficiency (LALD)Wolman disease
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