Cyclodextrin‐Based Macromolecular Systems as Cholesterol‐Mopping Therapeutic Agents in Niemann–Pick Disease Type C

Cyclodextrins (CDs) have gained considerable attention as a potential therapeutic intervention in the treatment of the rare genetic condition Niemann–Pick type C disease (NPC). However, in its monomeric form CDs suffer from serious side effects, especially from a pharmacokinetic and biodistribution standpoint. CD‐based macromolecular systems seem to be promising at overcome such limitations and might provide an improved therapeutic approach in cholesterol-associated diseases such as NPC. In this perspective article the latest developments in the preparation of CD‐containing polymers as cholesterol‐mopping therapeutic agents in NPC are summarized.