ABOUT MPS I DISEASE

Mucopolysaccharidosis type I (MPS I) is a rare lysosomal storage disease

MPS I is a rare, hereditary, potentially life-threatening disease that can affect multiple organ systems.

People with MPS I diseases do not make enough of an enzyme (lysosomal α-L-iduronidase) needed to break down long chains of sugar molecules. These chains of molecules are called glycosaminoglycans (GAGs, a type of mucopolysaccharide). The accumulated GAGs can cause heart and lung complications. These complications are the leading cause of early death in patients with MPS I.

How does MPS I disease affect one's body?

People with MPS I experience a wide range of symptoms and have different degrees of disease severity. MPS I is classified into 3 forms based on the severity of symptoms and rate of disease progression.

The Spectrum of MPS I

People with the most “severe” MPS I can suffer from symptoms that worsen over time, including cognitive impairment. People with “attenuated” MPS I – a milder form of MPS I – have limited cognitive impairment and growth deficiencies.