Infantile-onset Pompe disease entering adulthood: Insights from 2 decades of enzyme replacement therapy experience
婴儿期发病的庞贝病进入成年期：来自20年酶替代疗法经验的启示
期刊:Genetics in Medicine
影响因子:6.2
doi:10.1016/j.gim.2025.101590
Regmi, Neha; Kenney-Jung, Daniel; Stafford, Grace; Malinzak, Michael; Spiridigliozzi, Gail A; Boggs, Tracy; Koch, Rebecca L; Smith, Phillip Brian; Case, Laura E; Young, Sarah P; Jones, Harrison N; Kishnani, Priya S
