Elizabeth Berry-Kravis , Lisa A. LaGorio , Christine I. Dali , Daniel Gallo
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Qualitative assessment of the validity and standardization of the swallow domain in the 5-Domain Niemann-Pick disease type C (NPC) Clinical Severity Scale (5DNPCCSS) and analysis in an NPC clinical trial data set
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.