Targeted therapeutic benefits of anakinra for sleep, gastrointestinal, and behavioral symptoms in Sanfilippo syndrome: A post hoc analysis in symptom-specific subgroups
Jonathan Acevedo , Cara O'Neill , Adolfo Morales , Michelina Iacovino , Agnes Chen , Julie B. Eisengart , Lynda E. Polgreen
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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.