Monitoring and Assessments
Regular monitoring is an important part of MPS I management
Early diagnosis is critical to the early initiation of disease management.1
The Recommended Schedule of Assessments represents the core MPS I disease-related assessments that allow for evaluations of disease progression over time. Physicians will determine the actual frequency of necessary assessments according to a patient’s individualized need for medical care and routine
Minimum Recommended Schedule of Assessments for MPS I Patients
All tests requiring sedation are recommended only if sedation is considered to be safe for the patient.
*Diagnosis would include genotype, if available.
†May not be possible in non-cooperative patients or patients younger than 5 to 6 years of age.
‡The recommended method for obtaining organ volumes is MRI or computed tomography to enable quantitative analysis. However, if in the opinion of the clinician it is unsafe to sedate the patient, then ultrasound may be substituted.
§Measured in pediatric patients only, unless determined otherwise by treating physician.
These recommendations were developed by the MPS I Board of Advisors, a group of physicians who have experience in managing patients with MPS I. The MPS I Registry is sponsored and administered by Sanofi.
CNS, central nervous system; DQ, developmental quotients; ECG, electrocardiogram; FVC, forced vital capacity; FEV, forced expiratory volume; GAGs, glycosaminoglycans; IQ, intelligence quotient; MPS, mucopolysaccharidosis
Reference: 1. Pastores GM, Arn P, Beck M, et al. Mol Genet Metab. 2007;91(1):37-47.
MAT-US-2205121-v1.0-10/2022 Last Updated: October 2022