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

Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1

父母的困境:脊髓性肌萎缩症(SMA)1型患儿的治疗决策

Boursange, Sophie; Araneda, Marco; Stalens, Caroline; Desguerre, Isabelle; Barnerias, Christine; Nougues, Marie-Christine; Isapof, Arnaud; Quijano-Roy, Susana; Blu Genestine, Nadia; Ouillade, Laetitia; Martinez Jalilie, Maripaz; Castiglioni, Claudia; Boespflug-Tanguy, Odile; Gargiulo, Marcela

Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era

改善护理并赋能脊髓性肌萎缩症(SMA)成人患者:新治疗时代的行动呼吁

Walter, Maggie C; Chiriboga, Claudia; Duong, Tina; Goemans, Nathalie; Mayhew, Anna; Ouillade, Laëtitia; Oskoui, Maryam; Quinlivan, Ros; Vázquez-Costa, Juan F; Vissing, John; Servais, Laurent

Propelling Healthcare with Advanced Therapy Medicinal Products: A Policy Discussion

利用先进疗法药品推动医疗保健:政策探讨

Horgan, Denis; Metspalu, Andres; Ouillade, Marie-Christine; Athanasiou, Dimitrios; Pasi, John; Adjali, Oumeya; Harrison, Patrick; Hermans, Cedric; Codacci-Pisanelli, Giovanni; Koeva, Jasmina; Szucs, Thomas; Cursaru, Viorica; Belina, Ivica; Bernini, Chiara; Zhuang, Suijie; McMahon, Stephen; Toncheva, Draga; Thum, Thomas

Model consent clauses for rare disease research

罕见病研究的示范性知情同意条款

Nguyen, Minh Thu; Goldblatt, Jack; Isasi, Rosario; Jagut, Marlene; Jonker, Anneliene Hechtelt; Kaufmann, Petra; Ouillade, Laetitia; Molnar-Gabor, Fruszina; Shabani, Mahsa; Sid, Eric; Tassé, Anne Marie; Wong-Rieger, Durhane; Knoppers, Bartha Maria

Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for Patients

立场声明:共享脊髓性肌萎缩症临床研究数据,以加速研究并改善患者预后

Lochmüller, Hanns; Evans, David; Farwell, Wildon; Finkel, Richard; Goemans, Nathalie; de Lemus, Mencia; Matyushenko, Vitaliy; Muntoni, Francesco; Ouillade, Marie-Christine; Schwersenz, Inge; Wilson, Petra