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

Porter FD, Alexander DM, Albert OK, Robbins KP, Labor DA, Borruso AJ, Farhat NY, Jiang X, Berry-Kravis E

Open source

DOI
10.1016/j.ymgme.2025.109254
Published
2025 Dec
Container
Molecular genetics and metabolism
Publisher
Not recorded
Open access
yes

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BibTeX

@article{allodium:10.1016/j.ymgme.2025.109254,
  title = {Utility of 24(S)-hydroxycholesterol as a proximal biomarker to monitor long-term intrathecal adrabetadex therapy in individuals with Niemann-Pick disease, type C1.},
  author = {Porter FD and Alexander DM and Albert OK and Robbins KP and Labor DA and Borruso AJ and Farhat NY and Jiang X and Berry-Kravis E},
  year = {2025},
  journal = {Molecular genetics and metabolism},
  doi = {10.1016/j.ymgme.2025.109254},
  url = {https://doi.org/10.1016/j.ymgme.2025.109254}
}

RIS

TY  - JOUR
TI  - Utility of 24(S)-hydroxycholesterol as a proximal biomarker to monitor long-term intrathecal adrabetadex therapy in individuals with Niemann-Pick disease, type C1.
AU  - Porter FD
AU  - Alexander DM
AU  - Albert OK
AU  - Robbins KP
AU  - Labor DA
AU  - Borruso AJ
AU  - Farhat NY
AU  - Jiang X
AU  - Berry-Kravis E
PY  - 2025
JO  - Molecular genetics and metabolism
DO  - 10.1016/j.ymgme.2025.109254
UR  - https://doi.org/10.1016/j.ymgme.2025.109254
ER  - 

APA

FD, P., DM, A., OK, A., KP, R., DA, L., AJ, B., NY, F., X, J., & E, B. (2025). Utility of 24(S)-hydroxycholesterol as a proximal biomarker to monitor long-term intrathecal adrabetadex therapy in individuals with Niemann-Pick disease, type C1.. Molecular genetics and metabolism. https://doi.org/10.1016/j.ymgme.2025.109254

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