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

NEO1 Investigator Group

15 Citations (Scopus)
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Keyphrases

Pharmacology, Toxicology and Pharmaceutical Science

Immunology and Microbiology