Article
The natural history of MPS I: global perspectives from the MPS I Registry.
Genetics in medicine : official journal of the American College of Medical Genetics - 1 Oct 2014
Beck Michael, Arn Pamela, Giugliani Roberto, Muenzer Joseph, Okuyama Torayuki, Taylor John, Fallet Shari
Abstract excerpt
PURPOSE: In this study, we aimed to describe the natural history of mucopolysaccharidosis I. METHODS: Data from 1,046 patients who enrolled in the MPS I Registry as of August 2013 were available for descriptive analysis. Only data from untreated patients and data prior to treatment for patients who received treatment were considered. Age at symptom onset, diagnosis, and treatment initiation were examined by...
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