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sebelipase alfa (Kanuma)
Indications:
- lysosomal acid lipase deficiency
Adverse effects:
- nausea/vomiting, diarrhea, constipation
- rhinitis, cough
- fever
- anemia
- headache
Mechanism of action:
- recombinant form of the human lysosomal acid lipase produced in the egg whites of genetically engineered chickens
General
recombinant protein; chimer
metabolic agent (metabolic modifier)
References
- Medscape Oncology. Dec 8, 2015
FDA OKs First Drug (Kanuma) for Rare Enzyme Disease
http://www.medscape.com/viewarticle/855617