Short-Term Outcomes of Pediatric Patients With Mild Autosomal Recessive RPE65-Associated Retinal Dystrophy Treated With Voretigene Neparvovec
使用Voretigene Neparvovec治疗轻度常染色体隐性遗传RPE65相关视网膜营养不良患儿的短期疗效
期刊:Translational Vision Science & Technology
影响因子:2.6
doi:10.1167/tvst.14.8.8
Merle, David A; Hertens, Leen; Dimopoulos, Spyridon; Kohl, Susanne; Van Haute, Manon; De Baere, Elfride; De Bruyne, Marieke; Janssens, Barbara; Rüther, Klaus; Huchzermeyer, Cord; Mazzola, Pascale; Nerinckx, Fanny; Haack, Tobias; Wolfram, Lasse; Kempf, Melanie; Kühlewein, Laura; Stingl, Krunoslav; Leroy, Bart P; Stingl, Katarina