Barbara K. Burton , Bernd Schweikert , Olivia Okoli , Diana Sanchez-Hernandez , Jaco Botha , Jennifer Audi , Csaba Siffel , Olulade Ayodele
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Mucopolysaccharidosis type II sibling pairs study: A global retrospective chart review of effectiveness of idursulfase in infants and very young children with MPS II
期刊介绍:
Molecular Genetics and Metabolism contributes to the understanding of the metabolic and molecular basis of disease. This peer reviewed journal publishes articles describing investigations that use the tools of biochemical genetics and molecular genetics for studies of normal and disease states in humans and animal models.