Summary

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• Sebelipase alfa (Kanuma) is indicated for the treatment of patients diagnosed with Lysosomal Acid Lipase (LAL) deficiency, a rare genetic disorder caused by mutations in the LIPA gene.
• The information was derived from one systematic review/meta-analysis document that evaluated seven records across five individual studies involving 110 LAL-D patients.
• Significant improvements were observed in serum transaminases and lipid profiles, including total cholesterol, triglycerides, HDL-C, and LDL-C. No significant changes were noted in gamma-glutamyl transferase (GGT) or liver volume.
• Adverse events related to sebelipase alfa infusions were mostly mild to moderate in severity.
• Before sebelipase alfa's availability, treatments included dietary modifications and some lipid-modifying drugs which primarily controlled symptoms rather than addressing the underlying cause of LAL-D.
• The study population ranged from infants to adults with mean ages spanning from 2.57 months to 31.6 years; however, efficacy by specific subgroups such as disease severity or genetic variations was not detailed.