日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1

Nusinersen 与基因疗法在 1 型脊髓性肌萎缩症中的临床疗效比较

Ropars, Juliette; Cances, Claude; Garcia-Uzquiano, Rocio; Gomez-Garcia de la Banda, Marta; Barnerias, Christine; Audic, Frédérique; Durigneux, Julien; Halbert, Cécile; Nkam, Lionelle; Laugel, Vincent; Espil, Caroline; Walther-Louvier, Ulrike; Davion, Jean-Baptiste; Isapof, Arnaud; Le Goff, Laure; Desguerre, Isabelle; Quijano-Roy, Susana; Grimaldi, Lamiae

Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment

在脊髓性肌萎缩症1型患者中,onasemnogene abeparvovec单药治疗的真实世界多学科疗效:法国队列在治疗前三年的经验

Desguerre, Isabelle; Barrois, Rémi; Audic, Frédérique; Barnerias, Christine; Chabrol, Brigitte; Davion, Jean Baptiste; Durigneux, Julien; Espil-Taris, Caroline; Gomez-Garcia de la Banda, Marta; Guichard, Marine; Isapof, Arnaud; Nougues, Marie Christine; Laugel, Vincent; Le Goff, Laure; Mercier, Sandra; Pervillé, Anne; Richelme, Christian; Thibaud, Marie; Sarret, Catherine; Schweitzer, Cyril; Testard, Hervé; Trommsdorff, Valérie; Vanhulle, Catherine; Walther-Louvier, Ulrike; Altuzarra, Cécilia; Chouchane, Mondher; Ropars, Juliette; Quijano-Roy, Susana; Cances, Claude

ASC-1 Is a Cell Cycle Regulator Associated with Severe and Mild Forms of Myopathy

ASC-1 是一种细胞周期调节因子,与严重和轻微的肌病相关。

Villar-Quiles, Rocío N; Catervi, Fabio; Cabet, Eva; Juntas-Morales, Raul; Genetti, Casie A; Gidaro, Teresa; Koparir, Asuman; Yüksel, Adnan; Coppens, Sandra; Deconinck, Nicolas; Pierce-Hoffman, Emma; Lornage, Xavière; Durigneux, Julien; Laporte, Jocelyn; Rendu, John; Romero, Norma B; Beggs, Alan H; Servais, Laurent; Cossée, Mireille; Olivé, Montse; Böhm, Johann; Duband-Goulet, Isabelle; Ferreiro, Ana

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

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