Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial - Benedikt Schoser, Mark Roberts, Barry J. Byrne, Sheela Sitaraman, Hai Jiang, Pascal Laforêt, Antonio Toscano, Jeff Castelli, Jordi Díaz-Manera, Mitchell Goldman, Ans T. van der Ploeg, Drago Bratkovic, Srilakshmi Kuchipudi, Tahseen Mozaffar, Priya S. Kishnani - Dissemin

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Elsevier, The Lancet Neurology, 12(20), p. 1027-1037, 2021

DOI: 10.1016/s1474-4422(21)00331-8

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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

Journal article published in 2021 by Benedikt Schoser, Mark Roberts, Barry J. Byrne, Sheela Sitaraman, Hai Jiang, Pascal Laforêt, Antonio Toscano, Jeff Castelli, Jordi Díaz-Manera, Mitchell Goldman, Ans T. van der Ploeg, Drago Bratkovic, Srilakshmi Kuchipudi, Tahseen Mozaffar, Priya S. Kishnani and other authors.

This paper was not found in any repository, but could be made available legally by the author.

This paper was not found in any repository, but could be made available legally by the author.

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