Article
Clinical manifestations and treatment of mucopolysaccharidosis type I patients in Latin America as compared with the rest of the world.
Journal of inherited metabolic disease - 1 Oct 2011
Muñoz-Rojas María Verónica, Bay Luisa, Sanchez Luz, van Kuijck Marcel, Ospina Sandra, Cabello Juan Francisco, Martins Ana Maria
Abstract excerpt
BACKGROUND: Mucopolysaccharidosis I (MPS I) comprises a spectrum of clinical manifestations and is divided into three phenotypes reflecting clinical severity: Hurler, Hurler-Scheie, and Scheie syndromes. There may be important variations in clinical manifestations of this genetic disease in patients residing in different regions of the world. METHODS: Using data from the MPS I Registry (as of September 2009), we...
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