Pathogenicity of novel GLA gene missense mutations in Fabry disease and the therapeutic impact of migalastat.

Galafold (migalastat) - Amicus

http://www.ncbi.nlm.nih.gov/pubmed/40120803

Jingru Bi; Pengcheng Ji; Wenkai Guo; Yaqing Li; Yixuan Zhang; Yuansheng Xie

Mar 23, 2025