Trial in progress: An open-label study (AT1001-033; ASPIRE II) to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of migalastat in pediatric patients with Fabry disease

Molecular Genetics and Metabolism 2026

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Authors: Haichen Yang, Candice Lamb, Lee Ann Lawson, Elizabeth H. Moffat, Mary Padilla, Jennie Vosk, Franklin K. Johnson

DOI: 10.1016/j.ymgme.2025.109682

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Metabolism and Genetic Disorders