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To clarify the extent and chronology of surgical burden in relation to symptom onset and diagnosis in patients with mucopolysaccharidosis I (MPS I) as reported in the MPS I Registry, an international observational database.
Overall, the MPS I Registry database contains a broad sample of the global patient population, providing a potentially useful tool for expanding knowledge of MPS I and facilitating evidence-based decisions about the optimal means of monitoring and treating affected individuals.
Patient participation in the MPS I Registry is voluntary.
When interpreting results of data analyses from the MPS I Registry, certain limitations common to observational registries should be considered.
The MPS I Registry provides the largest global data set for evaluating the natural history, clinical presentation, and potential treatment response of patients with MPS I.
P.A. is the Chair of the North American Board of Advisors and a member of the International Board of Advisors for the MPS I Registry.
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The IAM CAT II-III registries were used to measure 30-day mortality for the pre-network period: IAM CAT II for patients treated with fibrinolysis and no reperfusion, and IAM CAT III for patients treated with primary PCI and rescue PCI.
"Is this registry just for your friends or your family, too?" "It's for everyone.
"So I understand [a registry] could truly frighten people and marginalize them so that they are disenfranchised".
"I started the registry mostly as a way to find my own friends, and now wanting to help others find theirs".
Until my four half-siblings sign up to the voluntary registry I am not allowed to know anything about them – no name, no basic information, no contact details, nothing.
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