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Mengel E, Patterson MC, Da Riol RM, et al. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021;44(6):1463-1480doi: 10.1002/jimd.12428.
Mengel, E., Patterson, M. C., Da Riol, R. M., Del Toro, M., Deodato, F., Gautschi, M., Grunewald, S., Grønborg, S., Harmatz, P., Héron, B., Maier, E. M., Roubertie, A., Santra, S., Tylki-Szymanska, A., Day, S., Andreasen, A. K., Geist, M. A., Havnsøe Torp Petersen, N., Ingemann, L., Hansen, T., Blaettler, T., Kirkegaard, T., & Í Dali, C. (2021). Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. Journal of inherited metabolic disease, 44(6), 1463-1480. https://doi.org/10.1002/jimd.12428
Mengel, Eugen, et al. "Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment." Journal of inherited metabolic disease vol. 44,6 (2021): 1463-1480. doi: https://doi.org/10.1002/jimd.12428
Mengel E, Patterson MC, Da Riol RM, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg S, Harmatz P, Héron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Andreasen AK, Geist MA, Havnsøe Torp Petersen N, Ingemann L, Hansen T, Blaettler T, Kirkegaard T, Í Dali C. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021 Nov;44(6):1463-1480. doi: 10.1002/jimd.12428. Epub 2021 Sep 07. PMID: 34418116.
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