Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale.

Mengel E, Patterson MC, Da Riol RM, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg SW, Harmatz P, Hennermann JB, Héron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, LaGorio L, Berry-Kravis E, Porter FD, Solomon B, Himmelstrup L, Mickle T, Guenther S, Dali CÍ

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DOI: 10.1016/j.ymgmr.2025.101233
Published: 2025 Jun
Container: Molecular genetics and metabolism reports
Publisher: Not recorded
Open access: yes

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Cite this work

BibTeX

@article{allodium:10.1016/j.ymgmr.2025.101233,
  title = {Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale.},
  author = {Mengel E and Patterson MC and Da Riol RM and Del Toro M and Deodato F and Gautschi M and Grunewald S and Grønborg SW and Harmatz P and Hennermann JB and Héron B and Maier EM and Roubertie A and Santra S and Tylki-Szymanska A and LaGorio L and Berry-Kravis E and Porter FD and Solomon B and Himmelstrup L and Mickle T and Guenther S and Dali CÍ},
  year = {2025},
  journal = {Molecular genetics and metabolism reports},
  doi = {10.1016/j.ymgmr.2025.101233},
  url = {https://doi.org/10.1016/j.ymgmr.2025.101233}
}

RIS

TY  - JOUR
TI  - Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale.
AU  - Mengel E
AU  - Patterson MC
AU  - Da Riol RM
AU  - Del Toro M
AU  - Deodato F
AU  - Gautschi M
AU  - Grunewald S
AU  - Grønborg SW
AU  - Harmatz P
AU  - Hennermann JB
AU  - Héron B
AU  - Maier EM
AU  - Roubertie A
AU  - Santra S
AU  - Tylki-Szymanska A
AU  - LaGorio L
AU  - Berry-Kravis E
AU  - Porter FD
AU  - Solomon B
AU  - Himmelstrup L
AU  - Mickle T
AU  - Guenther S
AU  - Dali CÍ
PY  - 2025
JO  - Molecular genetics and metabolism reports
DO  - 10.1016/j.ymgmr.2025.101233
UR  - https://doi.org/10.1016/j.ymgmr.2025.101233
ER  -

APA

E, M., MC, P., RM, D. R., M, D. T., F, D., M, G., S, G., SW, G., P, H., JB, H., B, H., EM, M., A, R., S, S., A, T., L, L., E, B., FD, P., B, S., L, H., T, M., S, G., & CÍ, D. (2025). Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale.. Molecular genetics and metabolism reports. https://doi.org/10.1016/j.ymgmr.2025.101233

Source records

pubmed · retrieved 2026-09-25T20:08:33.878Z