Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
一项法国真实世界观察研究:123名患有1型或2型脊髓性肌萎缩症(SMA)的儿童接受诺西那生治疗一年后的疗效
期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-020-01414-8
Audic, Frédérique; de la Banda, Marta Gomez Garcia; Bernoux, Delphine; Ramirez-Garcia, Paola; Durigneux, Julien; Barnerias, Christine; Isapof, Arnaud; Cuisset, Jean-Marie; Cances, Claude; Richelme, Christian; Vuillerot, Carole; Laugel, Vincent; Ropars, Juliette; Altuzarra, Cécilia; Espil-Taris, Caroline; Walther-Louvier, Ulrike; Sabouraud, Pascal; Chouchane, Mondher; Vanhulle, Catherine; Trommsdorff, Valérie; Pervillé, Anne; Testard, Hervé; Lagrue, Emmanuelle; Sarret, Catherine; Avice, Anne-Laude; Beze-Beyrie, Pierre; Pauly, Vanessa; Quijano-Roy, Susana; Chabrol, Brigitte; Desguerre, Isabelle