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

Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe

孤儿药认定指南:欧洲基因疗法的证据要求

Palomo, Gloria M; Pose-Boirazian, Tomas; Naumann-Winter, Frauke; Costa, Enrico; Duarte, Dinah M; Kalland, Maria E; Malikova, Eva; Matusevicius, Darius; Vitezic, Dinko; Larsson, Kristina; Magrelli, Armando; Stoyanova-Beninska, Violeta; Mariz, Segundo

Editorial: The changing focus of regulatory frameworks around the globe and the opportunities for harmonization

社论:全球监管框架的重点转变及其协调统一的机遇

Magrelli, Armando; O'Connor, Daniel J; Stoyanova-Beninska, Violeta

The Picture Interpretation Test 360°: A Virtual Reality Screening Tool for Executive Dysfunction and Rehabilitation Stratification in Mild Cognitive Impairment

图片解读测试360°:一种用于轻度认知障碍执行功能障碍和康复分层的虚拟现实筛查工具

Stramba-Badiale, Chiara; Noselli, Eleonora; Magrelli, Alessandra; Serino, Silvia; Pupillo, Chiara; De Gaspari, Stefano; Todisco, Sarah; Goulene, Karine; Stramba-Badiale, Marco; Tuena, Cosimo; Riva, Giuseppe

Revolutionizing CAR T-Cell Therapies: Innovations in Genetic Engineering and Manufacturing to Enhance Efficacy and Accessibility

革新CAR-T细胞疗法:基因工程和制造技术的创新以提高疗效和可及性

Giorgioni, Lorenzo; Ambrosone, Alessandra; Cometa, Maria Francesca; Salvati, Anna Laura; Nisticò, Robert; Magrelli, Armando

Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation

推进罕见病治疗:EMA十年来对罕见癌症工程化过继细胞疗法和孤儿药认定的深入研究

Kalland, Maria Elisabeth; Pose-Boirazian, Tomas; Palomo, Gloria Maria; Naumann-Winter, Frauke; Costa, Enrico; Matusevicius, Darius; Duarte, Dinah M; Malikova, Eva; Vitezic, Dinko; Larsson, Kristina; Magrelli, Armando; Stoyanova-Beninska, Violeta; Mariz, Segundo

Inherited retinal dystrophies and orphan designations in the European Union

欧盟的遗传性视网膜营养不良症和孤儿病认定

Moseley, Jane; Leest, Tim; Larsson, Kristina; Magrelli, Armando; Stoyanova-Beninska, Violeta

The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products

欧洲罕见病基因疗法现状:与欧洲药品管理局罕见病药品委员会合作六年的经验

Palomo, Gloria M; Pose-Boirazian, Tomas; Naumann-Winter, Frauke; Costa, Enrico; Duarte, Dinah M; Kalland, Maria E; Malikova, Eva; Matusevicius, Darius; Vitezic, Dinko; Larsson, Kristina; Magrelli, Armando; Stoyanova-Beninska, Violeta; Mariz, Segundo

CAR-T State of the Art and Future Challenges, A Regulatory Perspective

CAR-T 的现状和未来挑战,监管视角

Lorenzo Giorgioni, Alessandra Ambrosone, Maria Francesca Cometa, Anna Laura Salvati, Armando Magrelli

Licensing of Orphan Medicinal Products-Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021

孤儿药许可——2019年至2021年间欧洲药品管理局完成的上市许可申请中,真实世界数据和其他外部数据在疗效方面的应用

Naumann-Winter, Frauke; Wolter, Franziska; Hermes, Ulrike; Malikova, Eva; Lilienthal, Nils; Meier, Tania; Kalland, Maria Elisabeth; Magrelli, Armando

Real-World Data and Budget Impact Analysis (BIA): Evaluation of a Targeted Next-Generation Sequencing Diagnostic Approach in Two Orthopedic Rare Diseases

真实世界数据和预算影响分析 (BIA):评估针对两种骨科罕见疾病的下一代测序诊断方法

Elena Pedrini, Antonella Negro, Eugenio Di Brino, Valentina Pecoraro, Camilla Sculco, Elisabetta Abelli, Maria Gnoli, Armando Magrelli, Luca Sangiorgi, Americo Cicchetti