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All treatments Used in NPC care

Miglustat

An older substrate-reduction therapy that slows the buildup of certain lipids, and is now paired with Miplyffa.

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

How it works

Miglustat reduces the amount of certain fatty molecules the cell tries to process. If less lipid is made, less piles up inside the lysosome. It has been used for NPC for years and is part of the approved Miplyffa combination.

Who it is for

Often used with Miplyffa; historically used off-label or approved in some countries for NPC.

What it does not do

It does not restore NPC1 function or reverse damage that has already happened.

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

Too much lipid is heading into a blocked room

The lysosome cannot clear cholesterol and related fats. New lipids keep arriving anyway.

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.