Results 121 to 130 of about 125,197 (343)

Switching disease‐modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes

British Journal of Clinical Pharmacology, EarlyView.
With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić, Elvira Meni Maria Gkrinia, Patrick Eustaquio, Andrea Katrin Faour, Dinko Vitezić   +4 more
wiley   +1 more source

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

Author Correction: Antisense oligonucleotide development for the selective modulation of CYP3A5 in renal disease [PDF]

, 2021
Kevin A. Lidberg, Andrew J. Annalora, Marija Jozic, Daniel J. Elson, Lu Wang, Theo K. Bammler, Susanne Ramm, Maria Beatriz Monteiro, Jonathan Himmelfarb, Craig B. Marcus, Patrick L. Iversen, Edward J. Kelly   +11 more
openalex   +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

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

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

Antisense Oligonucleotide Technologies to Combat Obesity and Fatty Liver Disease [PDF]

, 2022
Michael F. Keating, Brian G. Drew, Anna C. Calkin   +2 more
openalex   +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

Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy [PDF]

, 2016
Accurate definition of genetic mutations causing Duchenne muscular dystrophy (DMD) has always been relevant in order to provide genetic counseling to patients and families, and helps to establish the prognosis in the case where the distinction between ...
Bello, Luca, Pegoraro, Elena
core