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THERAPIES

Mucopolysaccharidoses are rare genetic diseases that belong to the group of lysosomal storage diseases. There are six main types of MPS:
What all MPS diseases have in common is that a metabolic enzyme is not present or is insufficiently present. This leads to the deposition of glycosaminoglycans (GAG) in the lysosomes. The consequences are cell damage and functional restrictions of important organs as well as the excretion of GAG in the urine. In most cases the newborn MPS patients are still inconspicuous and only develop symptoms in the course of the first months of life.These are often unspecific in the first place
– but in their combination, they give an important indication of the disease.

NEWS

> Charitable access program
> International MPS day
> International MPS and related diseases symposium