Novel GLA variant in Fabry cardiomyopathy: evidence of pathogenicity and amenability to migalastat.

Galafold (migalastat) - BioMarin

http://www.ncbi.nlm.nih.gov/pubmed/42161426

Andi Xu; Chao Hu; Chenyu Cui; Fengnan Niu; Hui Li; Lian Wang; Xiaolei Zhu; Xue Zhang; Yingao Fan

May 21, 2026