AAV9-Mediated Gene Therapy for Infantile-Onset Pompe's Disease.
Ma X, Zhuang L, Ma W, Li J, Mao Y, Wang J, Wang X, Xu J, Yu S, Gu R, Wang Y, Li Z, Jiang X, Zhang S, He F, Yang X, Zhu L, Zhang S, Zhang Y, Li T, Li Q, Wu Z, Zhang C, Zhang Y, Dong X, Xiong H, Wu X, Feng Z.
Ma X, et al. Among authors: zhuang l.
N Engl J Med. 2025 Jun 26;392(24):2438-2446. doi: 10.1056/NEJMoa2407766.
N Engl J Med. 2025.
PMID: 40561529
Clinical Trial.