Arimoclomol for Niemann-Pick Disease type C - add-on therapy


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Interventions: Arimoclomol (BRX-345)
Indications: Niemann-Pick Disease
Year: 2017

Arimoclomol is a medicine that is under investigation as a treatment for NPC. It is given by mouth as capsules. Arimoclomol helps the body’s cells to process accumulated lipids. If licensed, arimoclomol could offer an additional treatment option for people with NPC.