Pablo Bianculli , Sefika Uslu Cil , Daniel Lewi , Kathleen Coolidge , Femida Gwadry-Sridhar , Antonio Oliveira-dos-Santos
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Prospective observational study to assess the long-term safety of olipudase alfa effect in pediatric patients less than 2 years of age with acid sphingomyelinase deficiency: Study design
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.