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All treatments Investigational

Cyclodextrin

A molecular sponge being studied for its ability to pull trapped cholesterol out of the lysosome.

Still image of Cyclodextrin
A still of the idea behind Cyclodextrin. The motion below is a simple animation, not a replacement for this picture.

How it works

Hydroxypropyl-beta-cyclodextrin can bind cholesterol. Researchers have studied whether it can enter cells and lift cholesterol out of overloaded lysosomes, like a sponge in a flooded room.

Forms such as adrabetadex and Trappsol Cyclo have been studied in trials. This is not a routine FDA-approved daily therapy for every person with NPC.

Who it is for

People in clinical trials or under specialist protocols, depending on the product and study.

What it does not do

It does not fix the broken gene. Access and evidence still depend on trial and regulatory status.

Step by step

What this treatment does

Four short pictures of the idea. Play through, or tap a step. This is a teaching picture, not a medical simulation.

Step 1 of 4 · The problem

Cholesterol is stuck inside the lysosome

NPC1 cannot move it out. The recycling room fills with gold-colored crystals of trapped fat.

Official information

Educational only. Ask an NPC specialist what is right for a specific child or adult. Official sites and FDA pages are the source of prescribing information.