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What is Sebelipase alfa
Sebelipase alfa is an intravenous therapy that provides a recombinant form of human lysosomal acid lipase (rhLAL) protein in patients with lysosomal acid lipase (LAL) deficiency.
LAL deficiency, also known as Wolman disease and cholesteryl ester storage disease (CESD), is an autosomal recessive lysosomal storage disorder that results in a marked decrease or loss in activity of the lysosomal acid lipase (LAL) enzyme.
Brand Name
Kanuma
Indications & Dosage
- lysosomal acid lipase (LAL) deficiency
Side Effects
- abdominal pain
- agitation
- anaphylactic shock
- anemia
- antibody formation
- anxiety
- asthenia
- chest pain (unspecified)
- chills
- constipation
- cough
- diarrhea
- dyspnea
- edema
- fever
- headache
- hyperemia
- hyperlipidemia
- hypertension
- hypotonia
- irritability
- nausea
- pallor
- pharyngitis
- pruritus
- rash
- rhinitis
- rhinorrhea
- sneezing
- tachypnea
- urticaria
- vomiting
Monitoring Parameters
- growth rate
- serum lipid profile
Contraindications
- breast-feeding
- egg hypersensitivity
- pregnancy
- requires a specialized care setting
Interactions
There are no drug interactions associated with Sebelipase alfa products.