Results 61 to 70 of about 12,767 (235)

A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases

Orphanet Journal of Rare Diseases
Background Real-world evidence (RWE) generated using real-world data (RWD) presents the potential to contextualize and/or supplement traditional clinical trials for regulatory approval of rare diseases (RDs).
Shailja Vaghela, Kaniz Afroz Tanni, Geetanjoli Banerjee, Vanja Sikirica   +3 more
doaj   +1 more source

Diffusion‐Based Generative Model With Scaffold‐Hopping Strategy Yields Highly Potent Bioactive Molecules

Advanced Science, EarlyView.
SMarT‐Diff introduces a multi‐objective generative paradigm that integrates scaffold hopping with structure‐aware scoring to enable controlled exploration beyond the training distribution. The framework consistently balances drug‐likeness, synthesizes accessibility and bioactivity, yielding chemically diverse candidates with enhanced properties.
Yuwei Yang, Xiaoqing Gong, Shukai Gu, Jing Li, Bo Liu, Yanan Tian, Qianqian Zhang, Xiaojun Yao, Huanxiang Liu   +8 more
wiley   +1 more source

Building learning health systems on Real World Data

International Journal of Integrated Care, 2019
Background: Health systems are witnessing two transitions that rarely look at each other: a) the epidemiological shift that implies health systems to adapt from the delivery of acute fragmented services to the provision of coordinated chronic care; and b)
Enrique Bernal-Delgado, Anibal García-Sempere, Ibai Tamayo-Rodríguez, Francisco Estupiñán-Romero, Felipe Aizupuru-Barandiaran, Ana Toledo-Chavarri   +5 more
doaj   +1 more source

Targeting PLD3 Reverses the Immunosuppressive Niche by Reprogramming Tumor‐Associated Macrophages and Potentiates Antitumor Immunity

Advanced Science, EarlyView.
PLD3 activates the lysosomal‐AKT‐NF‐κB axis to drive cellular senescence in macrophages, establishing an immunosuppressive TME by limiting the infiltration of cytotoxic T, NK, and NKT cells, which confers resistance to anti‐PD‐1 therapy. Abrine inhibits PLD3 expression, restoring antitumor immunity and synergizing with anti‐PD‐1 treatment.
Xingtu Qin, Qiong Li, Xuemei Xu, Yuhan Wu, Yin Zhu, Jieqiong Wu, Yuan Fang, Qiubei Chen, Shuyang Wang, Jiang Yu, Yaping Ye, Hongli Jiao   +11 more
wiley   +1 more source

A Brain‐Penetrant Nanobody Reveals GSK3β‐Driven Proline‐Directed Phosphorylation as a Master Regulator of Ischemic Neurodegeneration

Advanced Science, EarlyView.
A brain‐targeted nanoparticle enables delivery of a therapeutic nanobody (Nb.29E9) that inhibits pathogenic GSK3β signaling. This intervention restores AMPK/mTORC1/TGFβ homeostasis, attenuates neuroinflammation and oxidative stress, and promotes long‐term functional recovery after ischemic stroke.
Lan Li, Muyang Li, Lei Sun, Ying Yang, Yuanshun Wu, Ziyi Yin, Anni Wang, Peiyang Zhou, Shaoxiang Luo, Jian Chen, Jun Qin, Zhibing Ai, Zilong Yuan, Zhiqiang Dong, Min Zhang   +14 more
wiley   +1 more source

Impact of Delaying Disease Recurrence on Economic Burden in Patients with HER2+ Early-Stage Breast Cancer (eBC)

ClinicoEconomics and Outcomes Research
Nicole Princic,1 Eleanor Faherty,2 Meghan Moynihan,1 Caroline Henriques,2 Sandhya Mehta1 1Data Research & Analytics, Real World Data, MarketScan by Merative, Ann Arbor, MI, USA; 2HEOR & RWE, US Medical Affairs, Daiichi Sankyo, Inc., Basking Ridge,
Princic N, Faherty E, Moynihan M, Henriques C, Mehta S   +4 more
doaj  

Intrinsically Mitochondria‐Targeting Nanozyme via Coordination‐Assembly of Natural Quercetin for Cascade Antioxidant Therapy of Cerebral Ischemia‐Reperfusion Injury

Advanced Science, EarlyView.
This study uncovers that quercetin naturally targets mitochondria. By coordinating quercetin with Fe3+, we engineer an ultrasmall cascade nanozyme (MCN) with superoxide dismutase‐catalase activities. MCN crosses the damaged blood–brain barrier, scavenges mitochondrial ROS, prevents mitochondrial DNA leakage, and blocks the cGAS‐STING pathway, thereby ...
Wenxuan Zheng, Zhicheng Wang, Xin Zhou, Shuya Wang, Xiaojing Shi, Tingli Xiong, Ruishi Li, Yuting Lin, Zhen Chen, Jiawen Wei, Fei Li, Jinwen Ge, Kelong Ai, Chong Liu, Guiming Deng   +14 more
wiley   +1 more source

Expanded Hepatic Progenitor Cells Featured with Aggregation of α‐Synuclein Contribute to Pathologic Bile Duct Regeneration in Biliary Atresia

Advanced Science, EarlyView.
Expanded NCAM1+EpCAM+ hepatic progenitor cells in biliary atresia are characterized by aggregation of α‐synuclein. This pathological protein potentiates cellular susceptibility to GSH‐dependent redox dyshomeostasis, induces unstable biliary cell fate specification, and subsequently drives aberrant biliary regeneration.
Hua Xie, Mengyan Zhu, Zhongxian Zhu, Jiaqi Tang, Ruyi Zhang, Zequan Ding, Junzhi Li, Zhongluan Wu, Paul Kwong Hang Tam, Qianghu Wang, Vincent Chi Hang Lui, Yankai Xia, Weibing Tang   +12 more
wiley   +1 more source

Astrocytic FDX1 Contributes to Copper Dyshomeostasis‐associated Synaptic Dysfunction in Depression and Is Modulated by Exercise

Advanced Science, EarlyView.
Chronic stress disrupts copper homeostasis and promotes copper accumulation in the prelimbic cortex, leading to astrocytic FDX1 upregulation. Elevated astrocytic FDX1 impairs calcium signaling, induces structural atrophy, and disrupts synaptic function, contributing to depressive‐like behaviors. Physical exercise reverses these alterations by restoring
Lina Gao, Rongji Hui, Zhibo Tang, Tao Feng, Zhihang Hu, Xiaoqing Zhang, Ping Jiang, Hui Zhao, Kwok‐Fai So, Tianyuan Luo, Yanzhou Chang, Lan Yan   +11 more
wiley   +1 more source

A national evaluation analysis and expert interview study of real-world data sources for research and healthcare decision-making

Scientific Reports
Real-world data (RWD) can provide intel (real-world evidence, RWE) for research and development, as well as policy and regulatory decision-making along the full spectrum of health care.
Veronika Mikl, Dejan Baltic, Thomas Czypionka, Alexander Degelsegger-Márquez, Nikolaus Forgó, Ghazaleh Gouya-Lechner, Arnold Herzog, Peter Klimek, David Benjamin Lumenta, Bernhard Mraz, Herwig Ostermann, Robert Scharinger, Tanja Stamm, Michael Strassnig, Markus Zeitlinger, Johannes Pleiner-Duxneuner   +15 more
doaj   +1 more source