Can Alpha-Glucosidase Activity in Plasma or Leukocytes Serve as a Biomarker for Future Gene Therapy in Classic Infantile Pompe Disease?
血浆或白细胞中的α-葡萄糖苷酶活性能否作为经典婴儿型庞贝病未来基因治疗的生物标志物?
期刊:BioDrugs
影响因子:6.9
doi:10.1007/s40259-025-00757-7
Faraguna, Martha C; Lambregts, Daniël A M; Barzel, Ina; Jacobs, Ed H; Hoogeveen-Westerveld, Marianne; van der Beek, Nadine A M E; Pijnappel, W W M Pim; Gasperini, Serena; Preijers, Tim; van den Hout, Johanna M P; van der Ploeg, Ans T