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

AAV9 gene therapy optimization for SMARD1/CMT2S: safety and long-term efficacy comparison of two vectors in a SMARD1 preclinical model

针对SMARD1/CMT2S的AAV9基因治疗优化:在SMARD1临床前模型中比较两种载体的安全性和长期疗效

Pagliari, Elisa; Anastasia, Alessia; Bellandi, Floriana; Garbellini, Manuela; Ongaro, Jessica; Taiana, Michela; Comi, Giacomo P; Ottoboni, Linda; Sierra-Delgado, Julieth Andrea; Likhite, Shibi; Meyer, Kathrin C; Nizzardo, Monica; Corti, Stefania P

RNA biomarkers in spinal muscular atrophy: enhancing pathogenesis understanding and guiding precision medicine

脊髓性肌萎缩症中的RNA生物标志物:增进对发病机制的理解并指导精准医疗

Alberti, Claudia; Berardinelli, Angela; Comi, Giacomo P; Ottoboni, Linda; Corti, Stefania

Lewy pathology formation in patient-derived GBA1 Parkinson's disease midbrain organoids.

患者来源的GBA1帕金森病中脑类器官中路易病理的形成

Frattini Emanuele, Faustini Gaia, Lopez Gianluca, Carsana Emma Veronica, Tosi Mattia, Trezzi Ilaria, Magni Manuela, Soldà Giulia, Straniero Letizia, Facchi Daniele, Samarani Maura, Martá-Ariza Mitchell, De Luca Chiara Maria Giulia, Vezzoli Elena, Pittaro Alessandra, Stepanyan Astghik, Silipigni Rosamaria, Rosety Isabel, Schwamborn Jens C, Sardi Sergio Pablo, Moda Fabio, Corti Stefania, Comi Giacomo P, Blandini Fabio, Tritsch Nicolas X, Bortolozzi Mario, Ferrero Stefano, Cribiù Fulvia Milena, Wisniewski Thomas, Asselta Rosanna, Aureli Massimo, Bellucci Arianna, Di Fonzo Alessio

Opinion of the Italian Association of Myology on Ataluren for the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy

意大利肌病学会对阿塔卢伦治疗无义突变杜氏肌营养不良症的意见

Bello, Luca; Riguzzi, Pietro; Albamonte, Emilio; Astrea, Guja; Battini, Roberta; Barp, Andrea; Berardinelli, Angela L; Bertini, Enrico S; Brolatti, Noemi; Bruno, Claudio; Corti, Stefania; D'Amico, Adele; D'Angelo, Maria Grazia; Dallavalle, Gianfranco; Liguori, Rocco; Maggi, Lorenzo; Magri, Francesca; Mancuso, Michelangelo; Masson, Riccardo; Mercuri, Eugenio; Minetti, Carlo; Messina, Sonia; Mongini, Tiziana; Musumeci, Olimpia; Nigro, Vincenzo; Pane, Marika; Panicucci, Chiara; Pedemonte, Marina; Pini, Antonella; Politano, Luisa; Previtali, Stefano; Ricci, Federica; Ricci, Giulia; Ruggiero, Lucia; Sansone, Valeria; Siciliano, Gabriele; Trabacca, Antonio; Trucco, Federica; Velardo, Daniele; Pegoraro, Elena; Comi, Giacomo P

Unleashing the potential of mRNA therapeutics for inherited neurological diseases

释放mRNA疗法在遗传性神经系统疾病治疗中的潜力

Monfrini, Edoardo; Baso, Giacomo; Ronchi, Dario; Meneri, Megi; Gagliardi, Delia; Quetti, Lorenzo; Verde, Federico; Ticozzi, Nicola; Ratti, Antonia; Di Fonzo, Alessio; Comi, Giacomo P; Ottoboni, Linda; Corti, Stefania

Diffusion tensor imaging reveals subclinical alterations in muscles of patients with Becker muscular dystrophy

弥散张量成像揭示贝克尔肌营养不良症患者肌肉的亚临床改变

Nava, Simone; Conte, Giorgio; Triulzi, Fabio M; Comi, Giacomo P; Magri, Francesca; Velardo, Daniele; Cinnante, Claudia M

Myotonic Dystrophy type 2 unmasked by physical activity resumption following COVID-19 lockdown: case discussion and review of the literature

新冠疫情封锁后恢复体育活动揭示了2型强直性肌营养不良症:病例讨论及文献综述

Lucchiari, Sabrina; Magri, Francesca; Rimoldi, Martina; Pagliarani, Serena; Corti, Stefania; Comi, Giacomo P; Sciacco, Monica

Identification of Novel Biomarkers of Spinal Muscular Atrophy and Therapeutic Response by Proteomic and Metabolomic Profiling of Human Biological Fluid Samples

通过对人体生物体液样本进行蛋白质组学和代谢组学分析,鉴定脊髓性肌萎缩症及其治疗反应的新型生物标志物

Meneri, Megi; Abati, Elena; Gagliardi, Delia; Faravelli, Irene; Parente, Valeria; Ratti, Antonia; Verde, Federico; Ticozzi, Nicola; Comi, Giacomo P; Ottoboni, Linda; Corti, Stefania

Unraveling the Neurological Complexity of Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal Protein, and Skin Changes Syndrome: A Report of a Challenging Case of a Young Woman and Cutting-Edge Advancements in the Field

揭示多发性神经病、器官肿大、内分泌病、单克隆蛋白和皮肤改变综合征的神经系统复杂性:一例年轻女性的疑难病例报告及该领域的最新进展

Furciniti, Gioconda; Casalino, Giuseppe; Lo Russo, Francesco M; Bolli, Niccolò; Meneri, Megi; Comi, Giacomo P; Corti, Stefania P; Velardo, Daniele

Givinostat for Becker muscular dystrophy: A randomized, placebo-controlled, double-blind study

吉维诺司他治疗贝克尔型肌营养不良症:一项随机、安慰剂对照、双盲研究

Comi, Giacomo P; Niks, Erik H; Vandenborne, Krista; Cinnante, Claudia M; Kan, Hermien E; Willcocks, Rebecca J; Velardo, Daniele; Magri, Francesca; Ripolone, Michela; van Benthem, Jules J; van de Velde, Nienke M; Nava, Simone; Ambrosoli, Laura; Cazzaniga, Sara; Bettica, Paolo U