Results 161 to 170 of about 378,124 (311)

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

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

Synthesis of siRNAs containing carbocyclic nucleotides and the role of cyclopentane conformation in RNAi activity. [PDF]

RSC Chem Biol
Kundu J, Datta D, Akabane-Nakata M, Mandal S, Krampert M, Egli M, Manoharan M.   +6 more
europepmc   +1 more source

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source

Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe

British Journal of Clinical Pharmacology, EarlyView.
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis, Christine Leopold, Joyce M. Hoek, Raymond M. Schiffelers, Marie L. De Bruin   +4 more
wiley   +1 more source

The Gene Encoding the Antisense Protein ASP of HIV-1: Origin, Distribution and Maintenance. [PDF]

Viruses
Houmey MA, Sadek S, Daussy CF, Chazal N.
europepmc   +1 more source

Marine silicon for biomedical sustainability

BMEMat, EarlyView.
Schematic illustrating marine silicon for biomedical engineering. Abstract Despite momentous divergence from oceanic origin, human beings and marine organisms exhibit elemental homology through silicon utilization. Notably, silicon serves as a critical constituent in multiple biomedical processes.
Yahui Han, Zhibo Yang, Wei Xia, Chengtie Wu   +3 more
wiley   +1 more source

Guard cell photorespiration controls stomata behavior and development. [PDF]

New Phytol
Sun H, Thiemann I, Schmidt N, Kromdijk J, Lawson T, Hagemann M, Timm S.   +6 more
europepmc   +1 more source

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 reduction in NADP-ME in the C₄ species Flaveria bidentis alters stomatal sensitivity to intercellular [CO₂]. [PDF]

New Phytol
Bernardo EL, Sales CRG, Sun T, Kromdijk J.   +3 more
europepmc   +1 more source