Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study. [electronic resource]

By: Contributor(s): Producer: 20200601Description: 167-186 p. digitalISSN:
  • 1873-2364
Subject(s): Online resources: In: Neuromuscular disorders : NMD vol. 29
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Publication Type: Clinical Trial, Phase I; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't

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