Enzyme replacement therapy in adults with Muchopolysaccharidosis Type I

The journal Molecular Genetics and Metabolism has published an article about Muchopolysaccharidosis Type I. The efficacy of starting enzyme replacement therapy (ERT) in adults with Muchopolysaccharidosis Type I (MPS-I) is controversial. Evaluating the benefits reported by patients initiating ERT with laronidase at adult age might help physicians decide whether the use of ERT in these patients is worthwhile from a clinical point of view. The full-text article you can find here.