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Translational Dose-Response and Frequency Scaling for BMN 250 Administered Via the Intracerebroventricular Route: Predicting a Clinically Effective Dosing Regimen from Animal Models of Disease for the Treatment of Sanfilippo Syndrome Type B

Anita Grover,Emma McCullagh,Mika Aoyagi-Scharber,Stephen M. Maricich,Jill C. M. Wait,Andrew Melton,Huiyu Zhou,Roger Lawrence,Heather Prill,Brett Crawford,Joshua Henshaw

Molecular Genetics and Metabolism（2018）

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