Miglustat is the first and only specific drug approved for NPC in Europe, Canada and Japan. While Miglustat has shown to alleviate disease symptoms while attenuating neurodegeneration, some studies suggest its limited efficacy in NPC. In the US, the FDA declined its approval, requiring for more data. Hydroxypropyl-beta-cyclodextrin (HPβCD) has been used compassionately to treat patients with NPC and is undergoing clinical trials. HPβCD has shown to facilitate cholesterol transport inside of cells and its efflux. HPβCD is given to patients through either intrathecal or intravenous administration. Unfortunately, permanent hearing loss can occur following HPβCD therapy, with either route resulting in the preferential loss of cochlear outer hair cells (OHCs).