Arimoclomol is used alongside another medication to help manage neurological symptoms in children and adults with Niemann-Pick disease type C, a rare inherited condition where fatty substances build up in the body and damage the nervous system. The drug belongs to a class that activates heat shock proteins, though the exact mechanism of how it works is not fully understood.[1]
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Sep 20, 2024
MIPLYFFA
NDA NDA214927
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