DISEASE MONITORING AND ASSESSMENT
MPS I is a progressive disease
People with MPS I often show no symptoms at birth but develop signs and symptoms over time. Children with severe MPS I typically start to show symptoms within a year. Children with the attenuated form of MPS I can develop signs and symptoms after a few years.
Monitoring is important for managing disease progression
Because MPS I is progressive, it is important to monitor signs and symptoms over time by your doctor. Your doctor will decide what assessments and how often they need to be made based on your need for medical care.
Recommended schedule of assessments for patients with MPS I
Below are some possible assessments your doctor may want to repeat periodically. Please note that this is not a comprehensive list of all possible assessments for MPS I. Your doctor will decide based on your individual medical needs.
This recommended schedule is not a comprehensive list. Schedule of assessment may change due to the patient’s clinical presentation, please discuss with your healthcare team.
Self-monitoring
You can also monitor your, or your child’s, MPS I by keeping a note of any changes in your symptoms or if you experience any new ones.
Keeping track of symptoms can make it easier for your healthcare team to monitor your progress, both good and bad. Your healthcare team can decide if you’re staying on track or if any treatments are needed.
IQ, intelligence quotient; MPS I, mucopolysaccharidosis type I.