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Utility of 24(S)-hydroxycholesterol as a proximal biomarker to monitor long-term intrathecal adrabetadex therapy in individuals with Niemann-Pick disease, type C1

Number of pages: 7 Posted: 19 Sep 2025

Forbes Denny Porter, Derek M. Alexander, Orsolya Katalin Albert, Kendall P. Robbins, Desiree A. Labor, Aiden J. Borruso, Nicole M. Farhat, Xuntian Jiang and Elizabeth Berry-Kravis

Government of the United States of America - Eunice Kennedy-Shriver National Institutes of Child Health and Human Development, affiliation not provided to SSRN, Rush University - Rush University Medical Center, Rush University - Rush University Medical Center, affiliation not provided to SSRN, affiliation not provided to SSRN, affiliation not provided to SSRN, affiliation not provided to SSRN and Rush University - Rush University Medical Center

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Xuntian Jiang

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Scholarly Papers (1)

Utility of 24(S)-hydroxycholesterol as a proximal biomarker to monitor long-term intrathecal adrabetadex therapy in individuals with Niemann-Pick disease, type C1

Abstract: