Optimizing clinical outcomes: The journey of twins with CRIM-negative infantile-onset Pompe disease on high-dose enzyme replacement therapy and immunomodulation
优化临床疗效：接受高剂量酶替代疗法和免疫调节治疗的CRIM阴性婴儿型庞贝病双胞胎的治疗历程
期刊:Molecular Genetics and Metabolism Reports
影响因子:1.9
doi:10.1016/j.ymgmr.2024.101141
Fares, Angie H; Desai, Ankit K; Case, Laura E; Sharon, Cassie; Klinepeter, Amy; Kirby, Amelia; Lisi, Matthew T; Koch, Rebecca L; Kishnani, Priya S
