N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study.

Autor: Fields, T¹ (AUTHOR) tfields@intrabio.com, M. Bremova, T² (AUTHOR), Billington, I¹ (AUTHOR), Churchill, GC³ (AUTHOR), Evans, W^4,5 (AUTHOR), Fields, C¹ (AUTHOR), Galione, A³ (AUTHOR), Kay, R⁶ (AUTHOR), Mathieson, T^4,6 (AUTHOR), Martakis, K⁷ (AUTHOR), Patterson, M⁸ (AUTHOR), Platt, F³ (AUTHOR), Factor, M¹ (AUTHOR), Strupp, M⁹ (AUTHOR)

Publikováno v: Trials. 5/29/2023, Vol. 24 Issue 1, p1-9. 9p. 2 Diagrams, 2 Charts.

Late adult-onset Niemann Pick type C (NPC): An 'atypical' typical presentation at the age of 62

Autor: Sousa, M, Maamari, B, Bremova, T, Nuoffer, J M, Wiest, R, Amstutz, D, Krack, P, Bartholdi, D, Tinkhauser, G

Publikováno v: Sousa, M; Maamari, B; Bremova, T; Nuoffer, J M; Wiest, R; Amstutz, D; Krack, P; Bartholdi, D; Tinkhauser, G (2023). Late adult-onset Niemann Pick type C (NPC): An "atypical" typical presentation at the age of 62. (In Press). Parkinsonism & related disorders, p. 105460. Elsevier 10.1016/j.parkreldis.2023.105460

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::dc7785dd88c16427183187f9cbdbe330
https://boris.unibe.ch/184110/1/1-s2.0-S1353802023001839-main.pdf

Additional file 2 of N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study

Autor: Fields, T, M. Bremova, T, Billington, I, Churchill, GC, Evans, W, Fields, C, Galione, A, Kay, R, Mathieson, T, Martakis, K, Patterson, M, Platt, F, Factor, M, Strupp, M

Additional file 2: Supplementary Table 1. Parent Study schedule of enrolment, interventions, and assessments. Supplementary Table 2. Extension Phase schedule of enrolment, interventions, and assessments.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2a48036a62bde51d5e3e15c9edfad71e

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