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

More general terms

• neurologic agent

Database

• PubChem: http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?cid=70912