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

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.

AAV基因疗法治疗杜氏肌营养不良症:EMBARK 3期随机试验

Mendell Jerry R, Muntoni Francesco, McDonald Craig M, Mercuri Eugenio M, Ciafaloni Emma, Komaki Hirofumi, Leon-Astudillo Carmen, Nascimento Andrés, Proud Crystal, Schara-Schmidt Ulrike, Veerapandiyan Aravindhan, Zaidman Craig M, Guridi Maitea, Murphy Alexander P, Reid Carol, Wandel Christoph, Asher Damon R, Darton Eddie, Mason Stefanie, Potter Rachael A, Singh Teji, Zhang Wenfei, Fontoura Paulo, Elkins Jacob S, Rodino-Klapac Louise R

Exploring the natural history of bone mineral density in Duchenne muscular dystrophy: a systematic literature review

探索杜氏肌营养不良症患者骨矿物质密度的自然史:系统性文献综述

De Ford, Christian; Guridi, Maitea; Chen, Yingjia; Murphy, Alexander P; Wood, Claire; McMillan, Hugh J; Mercuri, Eugenio; Crabtree, Nicola; Ward, Leanne

The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy

Taldefgrobep Alfa 的临床开发:一种用于治疗杜氏肌营养不良症的抗肌肉生长抑制素 Adnectin

Muntoni, Francesco; Byrne, Barry J; McMillan, Hugh J; Ryan, Monique M; Wong, Brenda L; Dukart, Juergen; Bansal, Amita; Cosson, Valerie; Dreghici, Roxana; Guridi, Maitea; Rabbia, Michael; Staunton, Hannah; Tirucherai, Giridhar S; Yen, Karl; Yuan, Xiling; Wagner, Kathryn R

Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint

数字主要疗效终点首次监管资格认定的证据基础

Servais, Laurent; Strijbos, Paul; Poleur, Margaux; Mirea, Andrada; Butoianu, Nina; Sansone, Valeria A; Vuillerot, Carole; Schara-Schmidt, Ulrike; Scoto, Mariacristina; Seferian, Andreea M; Previtali, Stefano C; Tulinius, Már; Nascimento, Andrés; Furlong, Pat; Singh, Teji; Dreghici, Roxana Donisa; Goemans, Nathalie; Mercuri, Eugenio; Straub, Volker; Ormazabal, Maitea Guridi; Braid, Jessica; Muntoni, Francesco; Tricot, Alexis; Annoussamy, Mélanie; Eggenspieler, Damien

Stride Velocity 95th Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy

步速第95百分位指标比6分钟步行测试或北极星步行评估能在更短的时间间隔内检测到杜氏肌营养不良症患者的步行功能下降

Rabbia, Michael; Guridi Ormazabal, Maitea; Staunton, Hannah; Veenstra, Klaas; Eggenspieler, Damien; Annoussamy, Mélanie; Servais, Laurent; Strijbos, Paul

Stride Velocity 95th Centile: Insights into Gaining Regulatory Qualification of the First Wearable-Derived Digital Endpoint for use in Duchenne Muscular Dystrophy Trials

步速第95百分位:首个用于杜氏肌营养不良症试验的可穿戴设备衍生数字终点指标获得监管认证的见解

Servais, Laurent; Yen, Karl; Guridi, Maitea; Lukawy, Jacek; Vissière, David; Strijbos, Paul

The neuromuscular junction is a focal point of mTORC1 signaling in sarcopenia

神经肌肉接头是肌肉减少症中 mTORC1 信号传导的焦点

Daniel J Ham #, Anastasiya Börsch #, Shuo Lin, Marco Thürkauf, Martin Weihrauch, Judith R Reinhard, Julien Delezie, Fabienne Battilana, Xueyong Wang, Marco S Kaiser, Maitea Guridi, Michael Sinnreich, Mark M Rich, Nitish Mittal, Lionel A Tintignac, Christoph Handschin, Mihaela Zavolan #, Markus A Rüe

mTORC1 and PKB/Akt control the muscle response to denervation by regulating autophagy and HDAC4

mTORC1 和 PKB/Akt 通过调节自噬和 HDAC4 来控制肌肉对失神经支配的反应

Perrine Castets, Nathalie Rion, Marine Théodore, Denis Falcetta, Shuo Lin, Markus Reischl, Franziska Wild, Laurent Guérard, Christopher Eickhorst, Marielle Brockhoff, Maitea Guridi, Chikwendu Ibebunjo, Joseph Cruz, Michael Sinnreich, Rüdiger Rudolf, David J Glass, Markus A Rüegg

Alterations to mTORC1 signaling in the skeletal muscle differentially affect whole-body metabolism

骨骼肌中 mTORC1 信号通路的改变对全身代谢的影响各不相同

Guridi, Maitea; Kupr, Barbara; Romanino, Klaas; Lin, Shuo; Falcetta, Denis; Tintignac, Lionel; Rüegg, Markus A

Antioxidant peroxiredoxin 6 protein rescues toxicity due to oxidative stress and cellular hypoxia in vitro, and attenuates prion-related pathology in vivo

抗氧化过氧化物酶6蛋白可在体外挽救氧化应激和细胞缺氧引起的毒性,并在体内减轻朊病毒相关病理。

Asuni, Ayodeji A; Guridi, Maitea; Sanchez, Sandrine; Sadowski, Martin J