Results 31 to 40 of about 43,465 (252)

External validation of the IAIHG autoimmune hepatitis response criteria in a multicentric real-world cohort

JHEP Reports
Background & Aims: The goal of treatment in autoimmune hepatitis (AIH) is induction of remission to prevent the development of liver fibrosis, cirrhosis, and its related complications. Various definitions of treatment response and remission have been
Lorenz Grossar, Sarah Raevens, Christophe Van Steenkiste, Isabelle Colle, Charlotte De Vloo, Hans Orlent, Jeoffrey Schouten, Marie Gallant, Annelien Van Driessche, Sander Lefere, Lindsey Devisscher, Anja Geerts, Hans Van Vlierberghe, Xavier Verhelst   +13 more
doaj   +1 more source

What is the ideal endpoint in early-stage immunotherapy neoadjuvant trials in lung cancer?

Therapeutic Advances in Medical Oncology, 2023
Numerous clinical trials investigating neoadjuvant immune checkpoint inhibitors (ICI) have been performed over the last 5 years. As the number of neoadjuvant trials increases, attention must be paid to identifying informative trial endpoints.
Robert B. Cameron, Jacobi B. Hines, Valter Torri, Luca Porcu, Jessica Donington, Christine M. Bestvina, Everett Vokes, James M. Dolezal, Alessandra Esposito, Marina C. Garassino   +9 more
doaj   +1 more source

Validating ORR and PFS as surrogate endpoints in phase II and III clinical trials for NSCLC patients: difference exists in the strength of surrogacy in various trial settings

BMC Cancer, 2022
Objective This study aims to systematically validate the performance of surrogate endpoints in phase II and III clinical trials for NSCLC patients under various trial settings.
Tiantian Hua, Yuan Gao, Ruyang Zhang, Yongyue Wei, Feng Chen   +4 more
doaj   +1 more source

The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases

Orphanet Journal of Rare Diseases, 2011
Background Over 95% of rare diseases lack treatments despite many successful treatment studies in animal models. To improve access to treatments, the Accelerated Approval (AA) regulations were implemented allowing the use of surrogate endpoints to ...
Kakkis Emil D, Miyamoto Brigitta E
doaj   +1 more source

Comparing Biomarkers as Principal Surrogate Endpoints [PDF]

Biometrics, 2011
Recently a new definition of surrogate endpoint, the "principal surrogate," was proposed based on causal associations between treatment effects on the biomarker and on the clinical endpoint. Despite its appealing interpretation, limited research has been conducted to evaluate principal surrogates, and existing methods focus on risk models that consider
Huang, Ying, Gilbert, Peter B.
openaire   +3 more sources

Organ‐specific redox imbalances in spinal muscular atrophy mice are partially rescued by SMN antisense oligonucleotides

FEBS Letters, EarlyView.
We identified a systemic, progressive loss of protein S‐glutathionylation—detected by nonreducing western blotting—alongside dysregulation of glutathione‐cycle enzymes in both neuronal and peripheral tissues of Taiwanese SMA mice. These alterations were partially rescued by SMN antisense oligonucleotide therapy, revealing persistent redox imbalance as ...
Sofia Vrettou, Brunhilde Wirth
wiley   +1 more source

Tau acetylation at K331 has limited impact on tau pathology in vivo

FEBS Letters, EarlyView.
We mapped tau post‐translational modifications in humanized MAPT knock‐in mice and in amyloid‐bearing double knock‐in mice. Acetylation within the repeat domain, particularly around K331, showed modest increases under amyloid pathology. To test functional relevance, we generated MAPTK331Q knock‐in mice.
Shoko Hashimoto, Yukio Matsuba, Mika Takahashi, Takaomi C. Saido   +3 more
wiley   +1 more source

Surrogate endpoints for overall survival in digestive oncology trials: which candidates? A questionnaires survey among clinicians and methodologists

BMC Cancer, 2010
Background Overall survival (OS) is the gold standard for the demonstration of a clinical benefit in cancer trials. Replacement of OS by a surrogate endpoint allows to reduce trial duration.
Bonnetain Franck, Bedenne Laurent, Methy Nicolas   +2 more
doaj   +1 more source

An isoform of 14‐3‐3 protein regulates transbilayer lipid movement at the plasma membrane

FEBS Letters, EarlyView.
Loss of 14‐3‐3ζ in CHO cells confers resistance to exogenous phosphatidylserine (PS) and impairs endocytosis‐independent inward flip‐flop of fluorescent PS at the plasma membrane. RNAi‐mediated knockdown reproduces this defect, while no additive effect is seen in ATP11C‐deficient cells.
Akiko Yamaji‐Hasegawa, Akira Hattori, Masafumi Tsujimoto, Toshihide Kobayashi   +3 more
wiley   +1 more source

pH‐mediated activation of the lysosomal arginine sensor SLC38A9

FEBS Letters, EarlyView.
Cells monitor nutrient levels via the lysosomal transporter SLC38A9 to activate the mechanistic target of rapamycin complex 1 (mTORC1). This study reveals that SLC38A9 function is regulated by pH. We identified histidine 544 as a critical pH sensor that undergoes conformational changes to control amino acid efflux from lysosomes; therefore, it ...
Xuelang Mu, Ampon Sae Her, Tamir Gonen
wiley   +1 more source