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ARIMOCLOMOL IN ADULTS WITH NPC IN A REAL-WORLD SETTING DATA FROM AN EXPANDED ACCESS PROGRAM IN THE USA

Walla Al-Hertani,Elizabeth Berry-Kravis,Raymond Wang,Can Ficicioglu,Damara Ortiz,Caroline Hastings,Kristina Julich,Loren Pena, Paula Schleifer,Alexandra Ellis, Ronan O'Reilly, Meredith Hart,Daniel Gallo,Marc Patterson

Molecular Genetics and Metabolism（2022）

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