followME Fabry Pathfinders registry: 5-year cardiac, renal and multisystem effectiveness in patients on migalastat

Galafold (migalastat) - BioMarin

https://onlinelibrary.wiley.com/doi/epdf/10.1002/jimd.70230

Peter Nordbeck 1; Ales Linhart 2; Antonio Pisani 5; Olga Azevedo 4; Haichen Yang 5; Jennifer Hiros 5; Lee Ann Lawson 5; Vipul Jain 5; Biliana O Veleva-Rotse 5; Derralynn Hughes 6

Aug 17, 2026