Mucopolysaccharidosis 1 (MPS 1) Registry – Global Data Collection and Sharing of the Variability, Progression and Natural History of MPS1

Summary

An ongoing registry of disease outcomes for patients with MPS 1 disease.
The Registry is an international multi-center, strictly observational program for patients with MPS 1 Disease; no experimental intervention is involved. Thus, a patient will undergo clinical assessments and receive care as determined by the patient’s physician.
The goal of the MPS 1 Registry is to collect clinical data on people with MPS 1 disease around the world in an effort to help healthcare professionals involved in the treatment or diagnosis of MPS1 disease better understand it and its management.
The Registry database has been designed to collect a variety of baseline and follow-up data obtained through routine clinical and laboratory assessments. All data submission is voluntary and there are no predetermined follow-up periods. Participating sites are encouraged to enter comprehensive baseline data at enrollment and to perform follow-up assessments at regular intervals. While the protocol provides recommended schedules of Assessments each physician is solely responsible for determining the appropriate clinical care for each patient.