Results 201 to 210 of about 131,722 (303)

Olezarsen in Hypertriglyceridemia With High Cardiac Risk: A GRADE‐Assessed Meta‐Analysis of Randomized Trials With Trial Sequential Evidence

Endocrinology, Diabetes &Metabolism, Volume 9, Issue 3, May 2026.
This meta‐analysis of randomized trials evaluates olezarsen for hypertriglyceridemia in patients at high cardiovascular risk. Olezarsen significantly improved triglycerides and other atherogenic lipid parameters with generally acceptable safety, supporting its potential as a promising adjunct therapy while highlighting the need for larger and longer ...
Ahmed Emara, Ameer Awashra, Heba Aboeldahab, Ahmed Farid Gadelmawla, Hamza A. Abdul‐Hafez, Abdullah M. Alharran, Amal A. Alsubaiei, Muneera Jasim AlRumaihi, Sara Ahmed Albuhmaid, Abdalhakim Shubietah   +9 more
wiley   +1 more source

From Genomic Diagnosis to Personalized RNA Medicine: Advances in Next-Generation Sequencing and N-of-1 Antisense Oligonucleotide Therapies for Rare Genetic Diseases. [PDF]

Genes (Basel)
Rodriguez Carstens P, Moriyama H, Yokota T.   +2 more
europepmc   +1 more source

The Ubiquitin Ligase Zinc Finger SWIM Domain‐Containing Protein 8 Regulates Oligodendrocyte Development Through the Argonaute2/MicroRNA‐7 Axis

Glia, Volume 74, Issue 5, May 2026.
ZSWIM8, an E3 ubiquitin ligase that broadly targets IDR‐rich proteins, drives MiR‐7‐dependent AGO2 degradation and the turnover of many RBPs. Loss of ZSWIM8 leads to MiR‐7 accumulation in oligodendrocyte progenitor cells and myelination defects in the developing brain. ABSTRACT Proteostasis of proteins with intrinsically disordered regions (IDRs) is of
Jing Lei, Siming Zhong, Rong Fan, Xin Shu, Guan Wang, Jiansheng Guo, Shuting Xue, Luqian Zheng, Aiming Ren, Junfang Ji, Bing Yang, Shumin Duan, Zhiping Wang, Xing Guo   +13 more
wiley   +1 more source

Atrophin-1 antisense oligonucleotide provides robust protection from pathology in a fully humanized DRPLA model. [PDF]

Mol Ther Nucleic Acids
Smith VL, Gidi BZ, Bragg RM, Cantle JP, Ben-Varon A, Noble B, Prades S, Compton A, Greenfield J, Korecka JA, Gemos A, Yu T, Khurana V, Kordasiewicz HB, Zhao HT, Barker-Haliski M, Child DD, Carroll JB.   +17 more
europepmc   +1 more source

A Cationic Block Co‐Polymer for Gene Delivery in the Posterior Segment of the Eye

Journal of Biomedical Materials Research Part A, Volume 114, Issue 5, May 2026.
ABSTRACT Diseases of the back of the eye such as neovascular age‐related macular degeneration (nAMD) are vision threatening and treatment is burdensome for patients, often requiring ocular injections every other month. Injection risks and logistics lower patient compliance; however, even patients receiving optimal treatment can deteriorate.
Amber Monteiro, Odunayo O. Mugisho, Antonio de Souza, Lina Liu, Yosra Agban, Priyanka Agarwal, Avik Shome, Ilva D. Rupenthal, Heather Sheardown   +8 more
wiley   +1 more source

2025 Consensus Clinical Management Guidelines for Niemann‐Pick Disease Type C

Journal of Inherited Metabolic Disease, Volume 49, Issue 3, May 2026.
ABSTRACT In 2018, the International Niemann‐Pick Disease Alliance (INPDA) and the International Niemann‐Pick Disease Registry (INPDR) developed and published comprehensive clinical management guidelines to support inclusive and standardized care pathways in Niemann‐Pick disease type C (NPC)—an ultra‐rare, autosomal recessive, neurovisceral lysosomal ...
Tarekegn Hiwot, Forbes D. Porter, Tatiana Bremova‐Ertl, Uma Ramaswami, Caroline Hastings, Bénédicte Héron, Justin Hopkin, Joella Melville, Hernan Amartino, Mireia del Toro, Federica Deodato, Fatih Ezgü, Paul Gissen, James B. Gibson, Can Ficicioglu, Roberto Giugliani, Orna Staretz‐Chacham, Frances Platt, Nathalie Guffon, Kristina Julich, Nikola Kresojević, Anna Lehman, Yann Nadjar, Susanne A. Schneider, Simon Jones, Eugen Mengel, Michel Tchan, Mark Walterfang, Ozlem Goker‐Alpan, Charlotte Dawson, Sandra Cowie, Toni Mathieson, Elizabeth Berry‐Kravis, Marc C. Patterson   +33 more
wiley   +1 more source

Unifying the Communities of Early‐Onset Glycogen Storage Disease Type IV and Adult Polyglucosan Body Disease Through a Genetic Prevalence Study of GBE1‐Related Disease

JIMD Reports, Volume 67, Issue 3, May 2026.
Genetic prevalence study of glycogen storage disease type IV. In collaboration with the Rare Genomes Project at the Broad Institute of MIT and Harvard and the APBD Research Foundation, this study queried and curated variants in GBE1 from ClinVar, HGMD, and gnomAD to calculate the genetic prevalence of glycogen storage disease type IV (GSD IV).
Rebecca L. Koch, H. Orhan Akman, Erin Chown, Deberah Goldman, Jeff Levenson, Qing Lu, Lindsay T. Michalovicz Gill, Matthew Morgan, Jennifer L. Orthmann‐Murphy, Natacha T. Pires, Rebecca Reef, Harriet Saxe, Moriel Singer‐Berk, Samantha Baxter   +13 more
wiley   +1 more source

Safety, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide RO7239958 in healthy volunteers and adults with chronic hepatitis B infection. [PDF]

Antimicrob Agents Chemother
Geretti AM, Sostelly A, Buatois S, Lu S, Lemenuel A, Attley G, Bopst M, Alvarez-Sánchez R, Mueller H, Gane E.   +9 more
europepmc   +1 more source

Liver Fibrosis: Molecular Pathogenesis and Therapeutic Interventions

MedComm, Volume 7, Issue 5, May 2026.
We systematically summarized the etiologies, diagnostic approaches, and pathogenic mechanisms of liver fibrosis. Also, the therapeutic interventions for liver fibrosis were systematically classified into two main categories: etiological treatment and mechanism‐based antifibrotic therapies.
Jiaorong Qu, Wenqing Qin, Minghang Dong, Zhi Ma, Si Li, Runping Liu, Ranyun Chen, Changmeng Li, Xiaojiaoyang Li   +8 more
wiley   +1 more source