Safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from ASPIRE, a phase 3b, open-label, single-arm, 12-month clinical trial, and its open-label extension.

Galafold (migalastat) - Amicus

http://www.ncbi.nlm.nih.gov/pubmed/40215726

Amarilis Sanchez-Valle; Chester B Whitley; Damara Ortiz; Esperanza Font-Montgomery; Hai Jiang; Haichen Yang; Jennie Vosk; Lee Ann Lawson; Ozlem Goker-Alpan; Robert J Hopkin; Uma Ramaswami; William R Wilcox

Apr 12, 2025