Mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, is part of a family of lysosomal storage diseases called mucopolysaccharidoses.
These are genetic diseases that affect certain enzymes in the body that are found in lysosomes.
Proteins that facilitate a chemical reaction in the body, including breaking down other molecules.
Part of the cell that contains enzymes that break down complex molecules into smaller pieces that can be reused or recycled.
Proteins that facilitate a chemical reaction in the body, including breaking down other molecules.
Part of the cell that contains enzymes that break down complex molecules into smaller pieces that can be reused or recycled.
It’s important that heparan sulfate stays at certain levels in the body. When it builds up, its toxic effects on the cells of the brain and body lead to the progressive effects of MPS IIIA.
MPS IIIA occurs in fewer than
in the United States.*
*Data from 2017.
