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arimoclomol (Miplyffa)
Indications:
- Niemann-Pick disease type C
Dosage:
- oral capsules for use in combination with enzyme inhibitor miglustat
- 47-124 mg adminstered TID depending upon weight
Adverse effects:
- increase in serum creatinine of 10-20%
- most common adverse events in trials
- upper respiratory tract infection, diarrhea, weight loss
Mechanism of action:
- unknown
General
neurologic agent
Database Correlations
PUBCHEM correlations
References
- Lou N
First Drug Approved for Niemann-Pick Disease Type C.
Arimoclomol (Miplyffa) greenlit for ultra-rare genetic disorder.
MedPage Today September 20, 2024
https://www.medpagetoday.com/genetics/generalgenetics/112052