Current and emerging therapeutic advancements for Niemann-Pick disease type C: where are we now and what does the future hold?

Arimoclomol and NALL showed significant improvements of neurological symptoms and reasonable safety profiles in phase II/III trials. The recent approval of arimoclomol in combination with miglustat and NALL for patients with NPD type C has the potential to modify the standard of care and will hopefully improve the prognosis. However, the collection of real-world data is warranted to explore the real clinical value of these new medicaments. Cyclodextrins are also promising therapeutics that principally allow to correct basic pathophysiological mechanisms in NPD type C, but further research is needed.