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levacetylleucine (Aqneursa)
Indications:
- Niemann-Pick disease type C
Dosage:
- oral suspension
- one gram individual dose packet mixed in 40 mL of water, orange juice, or almond milk if taken by mouth
- mixed with water only if given through a G-tube
- administered orally up to three times daily with or without food
- dosage based on a patient's weight
Adverse effects:
- abdominal pain, dysphagia, upper respiratory tract infections, vomiting
Mechanism of action:
- unknown
General
neurologic agent
Database Correlations
PUBCHEM cid=70912
References
- Monaco K
Second Drug OK'd for Ultra-Rare Neurodegenerative Disease.
FDA approves levacetylleucine (Aqneursa) for Niemann-Pick disease type C.
MedPage Today September 25, 2024
https://www.medpagetoday.com/genetics/generalgenetics/112111