Results 111 to 120 of about 117,595 (294)

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

British Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić, Ivana Stević, Elvira Meni Maria Gkrinia, Dinko Vitezić, Slobodan Janković   +4 more
wiley   +1 more source

Tissue pharmacokinetics of antisense oligonucleotides

Molecular Therapy: Nucleic Acids
Pharmacokinetics (PK) of antisense oligonucleotides (ASOs) is characterized by rapid distribution from plasma to tissue and slow terminal plasma elimination driven by re-distribution from tissue.
Erica Bäckström, Alessandro Bonetti, Per Johnsson, Stefan Öhlin, Anders Dahlén, Patrik Andersson, Shalini Andersson, Peter Gennemark   +7 more
doaj   +1 more source

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, EarlyView.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Antisense Oligonucleotide Therapy for Calmodulinopathy

Circulation
BACKGROUND: Calmodulinopathies are rare inherited arrhythmia syndromes caused by dominant heterozygous variants in CALM1 , CALM2 , or CALM3 , which each encode the identical CaM (calmodulin) protein. We hypothesized that antisense
Raul H. Bortolin, Farina Nawar, Chaehyoung Park, Michael A. Trembley, Maksymilian Prondzynski, Mason E. Sweat, Peizhe Wang, Jiehui Chen, Fujian Lu, Carter Liou, Paul Berkson, Erin M. Keating, Daisuke Yoshinaga, Nikoleta Pavlaki, Thomas Samenuk, Cecilia B. Cavazzoni, Peter T. Sage, Qing Ma, Robert D. Whitehill, Dominic J. Abrams, Chrystalle Katte Carreon, Juan Putra, Sanda Alexandrescu, Shuai Guo, Wen-Chin Tsai, Michael Rubart, Dieter A. Kubli, Adam E. Mullick, Vassilios J. Bezzerides, William T. Pu   +29 more
openaire   +2 more sources

Vacancy defect‐induced electron homing breaks phosphodiester bonds for RNA depletion‐driven cancer therapy

BMEMat, EarlyView.
Illustration of 5% S‐vacancy Bi2S3 mediated phosphodiester bonds cleavage in RNA of hepatocellular carcinoma cells, which suppressing ERI3 expression, inhibiting cell proliferation and promoting apoptosis. Abstract Genome‐wide hypertranscription is a hallmark of malignant progression.
Chuncheng Yang, Zehao Shen, Guangming Xiang, Zhe Liu, Huaxin Liu, Jiabei Li, Ao Wang, Yelin Wu, Peiran Zhao, Xingwu Jiang, Jinghan Wang, Xiaoqing Jiang, Wenbo Bu   +12 more
wiley   +1 more source

Boron Neutron Capture Therapy at a Crossroads: Translational Gap and Emerging Delivery Agents

Chemistry – A European Journal, EarlyView.
This review surveys recent advances in boron delivery agents for BNCT, emphasizing the shift from classical small molecules to multifunctional nanocarriers and theranostic systems. By integrating targeting, imaging, and therapy, next‐generation boron compounds aim to bridge the gap between (bio)chemical innovation and clinical translation.
Christoph Selg, Evamarie Hey‐Hawkins
wiley   +1 more source

Self‐Immolative Gels: Programmable Degradation Using Self‐Immolative Linkers and Polymers

Chemistry – A European Journal, EarlyView.
Self‐immolative linkers and polymers undergo bond‐cleaving reaction cascades in response to specific stimuli. Their incorporation into gels can be harnessed to release cargo, induce property changes, or trigger degradation in a highly controlled manner. These functions can be employed in applications such as sensing and drug delivery.
Chuanfeng Li, Elizabeth R. Gillies
wiley   +1 more source

Role of SoxE transcription factors in development and disease

Developmental Dynamics, EarlyView.
Abstract Sox8, Sox9, and Sox10 arose by multiple rounds of genome duplications from a single SoxE gene in ancestral vertebrates. In this review, we will briefly discuss the molecular structure and function of SoxE transcription factors and their evolutionary origin. We will then discuss their expression, function, and developmental disorders.
Merin Lawrence, Gerhard Schlosser
wiley   +1 more source

Evolution of the European Medicines Agency clinical guidelines for epilepsy drug development between 2010 and 2025: A comparative analysis by the ILAE Task Force on Regulatory Affairs

Epilepsia, EarlyView.
Abstract Objective The latest European Medicines Agency (EMA) guideline on the clinical investigation of medicines to treat epileptic disorders was adopted by the EMA Committee for Medicinal Products for Human Use in 2025. We compared this guideline with the previous version (2010), highlighting areas where significant revisions were introduced ...
Stéphane Auvin, Alexis Arzimanoglou, Jacqueline French, Kelly G. Knupp, Lieven Lagae, Eugen Trinka, Dennis Dlugos, Emilio Perucca   +7 more
wiley   +1 more source

Decrease in gyrase A protein expression in _E. coli_ cells inhibited by antisense ribozymes [PDF]

, 2008
RNase P complexed with external guide sequence (EGS) represents a novel nucleic-acid-based gene interference approach to modulate gene expression. Nucleic acid-based gene interference technologies represent promising strategies for specific inhibition of
Malathi Raghunathan, Shilpakala Sainath Rao   +1 more
core   +1 more source