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{"title":"UV1 vaccination in pembrolizumab-treated patients with recurrent or metastatic head and neck cancer: A randomized multicenter phase 2 trial.","authors":"Anna Brandt, Konrad Klinghammer, Christoph Schultheiss, Lisa Paschold, Claudia Wickenhauser, Marcus Bauer, Anna Bergqvist, Dennis Hahn, Philippe Schafhausen, Mareike Tometten, Markus Blaurock, Henrike Barbara Zech, Chia-Jung Busch, Andreas Dietz, Urs Müller-Richter, Jürgen Alt, Andreas Boehm, Simone Kowoll, Jörg Steighardt, Alexander Lasch, Ingunn Hagen Westgaard, Marita Westhrin, Alexander Stein, Axel Hinke, Mascha Binder","doi":"10.1016/j.medj.2025.100647","DOIUrl":"10.1016/j.medj.2025.100647","url":null,"abstract":"<p><strong>Background: </strong>Human telomerase reverse transcriptase is highly expressed in head and neck squamous cell carcinoma (HNSCC). The FOCUS study examines the role of the telomerase-directed vaccine UV1 in combination with pembrolizumab in patients with recurrent or metastatic (R/M) HNSCC.</p><p><strong>Methods: </strong>The FOCUS trial, a two-armed, open-label, randomized, multicenter phase 2 study, was designed to assess the efficacy and feasibility of UV1 as an add-on to pembrolizumab in the first-line treatment of patients with R/M PD-L1⁺ HNSCC. A progression-free survival (PFS) rate at 6 months of 40% was deemed promising for further development in a phase 3 setting. The trial was conducted in 10 centers in Germany (this study was registered at ClinicalTrials.gov: NCT05075122).</p><p><strong>Findings: </strong>From August 2021 to July 2023, 25 patients were enrolled in the pembrolizumab arm and 50 patients in the pembrolizumab + UV1 arm. The PFS rate at 6 months was 40% in the pembrolizumab arm and 30% in the pembrolizumab + UV1 arm. No specific safety signals were observed in the pembrolizumab + UV1 arm apart from a reversible allergic reaction that appeared in one patient. At a median follow-up of 11.3 months, median overall survival was 13.1 months in the pembrolizumab arm and 12.6 months in the pembrolizumab + UV1 arm.</p><p><strong>Conclusions: </strong>The addition of UV1 to pembrolizumab was safe but did not show an efficacy signal in this study population.</p><p><strong>Funding: </strong>The legal sponsor of the trial was the University Medical Center Halle (Saale), Germany and was funded by a grant from Ultimovacs.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100647"},"PeriodicalIF":12.8,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144031625","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"OnSIDES database: Extracting adverse drug events from drug labels using natural language processing models.","authors":"Yutaro Tanaka, Hsin Yi Chen, Pietro Belloni, Undina Gisladottir, Jenna Kefeli, Jason Patterson, Apoorva Srinivasan, Michael Zietz, Gaurav Sirdeshmukh, Jacob Berkowitz, Kathleen LaRow Brown, Nicholas P Tatonetti","doi":"10.1016/j.medj.2025.100642","DOIUrl":"10.1016/j.medj.2025.100642","url":null,"abstract":"<p><strong>Background: </strong>Adverse drug events (ADEs) are the fourth leading cause of death in the US and cost billions of dollars annually in increased healthcare costs. However, few machine-readable databases of ADEs exist, limiting our capacity to study drug safety on a broader, systematic scale. Recent advances in natural language processing methods, such as BERT models, present an opportunity to accurately extract relevant information from unstructured biomedical text.</p><p><strong>Methods: </strong>We fine-tune a PubMedBERT model to extract ADE terms from text in FDA Structured Product Labels for prescription drugs. Here, we present OnSIDES (on-label side effects resource), a compiled, machine-friendly database of drug-ADE pairs generated with this method. We further utilize this method to extract pediatric-specific ADEs, serious ADEs from labels' \"Boxed Warnings\" section, and ADEs from drug labels of other major nations-the UK, the European Union, and Japan-to build a complementary OnSIDES-INTL database. To present OnSIDES' potential applications, we leverage the database to predict novel drug targets and indications, analyze enrichment of ADEs across drug classes, and predict novel ADEs from chemical compound structures.</p><p><strong>Findings: </strong>We achieve an F1 score of 0.90, AUROC of 0.92, and AUPR of 0.95 at extracting ADEs from the labels' \"Adverse Reactions\" section. OnSIDES contains over 3.6 million drug-ADE pairs for 3,233 unique drug ingredient combinations extracted from 47,211 labels.</p><p><strong>Conclusions: </strong>OnSIDES can be used as a comprehensive resource to study and enhance drug safety.</p><p><strong>Funding: </strong>R35GM131905 to N.P.T.; T32GM145440 to H.Y.C.; and T15LM007079 to U.G., M.Z., and K.L.B.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100642"},"PeriodicalIF":12.8,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12256195/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143781524","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Early-warning signals and the role of H9N2 in the spillover of avian influenza viruses.","authors":"Yan-He Wang, Jin-Jin Chen, Jun Ma, Jonathan E Owen, Guo-Lin Wang, Lin-Jie Yu, Chun-Xi Shan, Yao Tian, Chen-Long Lv, Tao Wang, Yan Zhang, Sheng-Hong Lin, Xin-Jing Zhao, Sheng Zhang, Wang-Qian Wei, Yuan-Yuan Zhang, Tian Tang, Xin-Lou Li, Tao Jiang, Jing Li, Xiao-Ai Zhang, Feng Hong, Simon I Hay, Yan-Song Sun, Wei Liu, Li-Qun Fang","doi":"10.1016/j.medj.2025.100639","DOIUrl":"10.1016/j.medj.2025.100639","url":null,"abstract":"<p><strong>Background: </strong>The spillover of avian influenza viruses (AIVs) presents a significant global public health threat, leading to unpredictable and recurring pandemics. Current pandemic assessment tools suffer from deficiencies in terms of timeliness, capability for automation, and ability to generate risk estimates for multiple subtypes in the absence of documented human cases.</p><p><strong>Methods: </strong>To address these challenges, we created an integrated database encompassing global AIV-related data from 1981 to 2022. This database enabled us to estimate the rapid expansion of spatial range and host diversity for specific AIV subtypes, alongside their increasing prevalence in hosts that have close contact with humans. These factors were used as early-warning signals for potential AIV spillover. We analyzed spillover patterns of AIVs using machine learning models, spatial Durbin models, and phylogenetic analysis.</p><p><strong>Findings: </strong>Our results indicate a high potential for future spillover by subtypes H3N1, H4N6, H5N2, H5N3, H6N2, and H11N9. Additionally, we identified a significant risk for re-emergence by subtypes H5N1, H5N6, H5N8, and H9N2. Furthermore, our analysis highlighted 12 key strains of H9N2 as internal genetic donors for human adaptation in AIVs, demonstrating the crucial role of H9N2 in facilitating AIV spillover.</p><p><strong>Conclusions: </strong>These findings provide a foundation for rapidly identifying high-risk subtypes, thus optimizing resource allocation in vaccine manufacture. They also underscore the potential significance of reducing the prevalence of H9N2 as a complementary strategy to mitigate chances of AIV spillovers.</p><p><strong>Funding: </strong>National Key Research and Development Program of China.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100639"},"PeriodicalIF":12.8,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143731923","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Chromatin accessibility states affect transrenal clearance of plasma DNA: Implications for urine-based diagnostics.","authors":"Mary-Jane L Ma, Woody Z Zhang, Peiyong Jiang, Lu Ji, Dongyan Xiong, Wenlei Peng, W K Jacky Lam, Stephanie C Y Yu, L Y Lois Choy, Ryan Tsz-Hei Tse, Suk Hang Cheng, Qing Zhou, Jinyue Bai, Xi Hu, Yuwei Shi, Landon L Chan, W T Charlotte Chan, Pik-Ying Wong, Sherwood Fung, So Ling Lau, John Wong, Stephen L Chan, Peter K F Chiu, Jeremy Y C Teoh, Liona C Poon, Chi-Fai Ng, Cheuk-Chun Szeto, K C Allen Chan, Y M Dennis Lo","doi":"10.1016/j.medj.2025.100646","DOIUrl":"10.1016/j.medj.2025.100646","url":null,"abstract":"<p><strong>Background: </strong>Urinary cell-free DNA (ucfDNA) is a valuable resource for truly non-invasive liquid biopsy. UcfDNA comprises transrenal ucfDNA passing from the bloodstream through the glomeruli and locally shed urinary-tract ucfDNA. Understanding their differences in characteristics may enable new diagnostic applications.</p><p><strong>Methods: </strong>We analyzed 136 ucfDNA samples from healthy controls, pregnant women, patients with chronic kidney diseases (CKDs), and bladder cancer using massively parallel sequencing. Fragmentomic characteristics including fragment sizes and 5' end motifs were deduced. The relationship between ucfDNA and chromatin accessibility was examined by overlapping ucfDNA with open chromatin regions (OCRs, lacking histones) and heterochromatin regions (HCRs, tightly packed with histones).</p><p><strong>Findings: </strong>Compared with urinary-tract ucfDNA, the transrenal ucfDNA was shorter and enriched for C-ends. The transrenal ucfDNA was over-represented in OCRs but depleted in HCRs, indicating an interplay between the glomerular filtration barrier and the effective cfDNA size. In patients with proteinuria (preeclampsia and CKDs), the amount of ucfDNA from HCRs increased, suggesting elevated glomerular permeability of histone-bound plasma DNA molecules. In oncology, the use of hypomethylation signals in HCRs enhanced bladder cancer detection, with an area under the receiver operating characteristic curve of 0.93.</p><p><strong>Conclusions: </strong>Chromatin accessibility states impact the transrenal clearance of plasma DNA, likely through the size restriction of the glomerular barrier. This realization has enabled the rational development of novel approaches for detecting or monitoring renal dysfunction and urological cancers.</p><p><strong>Funding: </strong>The Innovation and Technology Commission of the Hong Kong SAR Government (InnoHK initiative) and the Li Ka Shing Foundation supported this study.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100646"},"PeriodicalIF":12.8,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144049848","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tolebrutinib for non-relapsing secondary progressive multiple sclerosis: a critical therapeutic gap.","authors":"Maria A Rocca, Paolo Preziosa, Massimo Filippi","doi":"10.1016/j.medj.2025.100751","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100751","url":null,"abstract":"<p><p>Bruton tyrosine kinase inhibitors (BTKi) represent a promising strategy to limit disability accumulation in multiple sclerosis (MS). Results from the HERCULES trial¹ showed that tolebrutinib, a brain-penetrant BTKi, reduced by 31% the risk of disability progression in non-relapsing secondary progressive MS patients, with a good safety profile. Tolebrutininb may represent a major advance for this MS population with no treatment currently approved.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":"6 7","pages":"100751"},"PeriodicalIF":12.8,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144620810","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Plinabulin following radiation enhances dendritic cell maturation and checkpoint inhibitor retreatment of relapsed/refractory cancers.","authors":"Steven H Lin, Vivek Subbiah, Evan N Cohen, Ziyi Li, Yingjuan June Lu, Ye Lin Son, Yue Lyu, Hui Gao, Gitanjali Jayachandran, Shinya Neri, Amrish Sharma, Penny Fang, Daniel Karp, David Hong, Jordi Rodon, Hao Yu, Jing Peng, G Kenneth Lloyd, James R Tonra, James M Reuben, Lan Huang, Siqing Fu","doi":"10.1016/j.medj.2025.100752","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100752","url":null,"abstract":"<p><strong>Background: </strong>Plinabulin exerts immunomodulatory activity through guanine nucleotide exchange factor (GEF)-H1 release from depolymerizing tubulin in the cytoskeleton, leading to dendritic cell (DC) activation. Preclinical studies demonstrated that irradiation potentiates plinabulin-induced DC maturation and, when combined with immune checkpoint inhibitors (ICIs), triggers an abscopal antitumor response via increased tumor-infiltrating DCs and T cells.</p><p><strong>Methods: </strong>A phase 1 translational study (NCT04902040) of plinabulin plus ICIs after radiation therapy (RT) initiation was conducted in ICI-relapsed/refractory cancers with primary (safety, tolerability, and objective tumor response rate) and secondary (disease control rate [DCR]) endpoints.</p><p><strong>Findings: </strong>This triple regimen was safe and achieved a DCR of 54% (3/13 partial response [PR] and 4/13 stable disease [SD]) in mostly heavily pretreated patients. Responding tumors included non-small cell lung cancer (2/2 PR + SD), head-and-neck squamous cell carcinoma (2/3 PR + SD), and Hodgkin's lymphoma (2/2 PR in patients after 12 or 16 prior lines of therapy). PR + SD patients had significantly higher GEF-H1 immune-activation scores in peripheral blood and intratumorally at pretreatment/baseline and DC activation/T cell clonal expansion post-treatment compared with progressive disease patients.</p><p><strong>Conclusions: </strong>These preliminary results provide a rationale for testing RT/plinabulin/ICI combination in future post-ICI-failure confirmatory trials.</p><p><strong>Funding: </strong>This study was funded by BeyondSpring Pharmaceuticals, Inc.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100752"},"PeriodicalIF":12.8,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144529956","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A phase 3, randomized trial to evaluate lot-to-lot consistency of V116, an adult-specific pneumococcal conjugate vaccine (STRIDE-4).","authors":"Paul Scott, Benita Ukkonen, Yoseph Caraco, Silvia Narejos Perez, Sady Armada Alpizar, Jose Francisco Cardona, David Greenberg, Carlos G Grijalva, Walter Orenstein, Richard T Wiedmann, Doreen Fernsler, Kyeongmi Cheon, Jianing Li, Heather Loryn Platt","doi":"10.1016/j.medj.2025.100748","DOIUrl":"10.1016/j.medj.2025.100748","url":null,"abstract":"<p><strong>Background: </strong>Streptococcus pneumoniae infection can lead to community-acquired pneumonia and invasive pneumococcal disease (IPD), conditions associated with substantial morbidity and mortality. V116 (Merck & Co., Inc., Rahway, NJ, USA) is a 21-valent, adult-specific pneumococcal conjugate vaccine (PCV) indicated for protection against pneumonia and IPD caused by S. pneumoniae.</p><p><strong>Methods: </strong>This global phase 3 trial (ClinicalTrials.gov: NCT05464420) evaluated the manufacturing consistency of V116. Adult participants were randomly assigned to receive a single dose of V116 from one of three lots or the 23-valent pneumococcal polysaccharide vaccine (PPSV23). Serotype-specific opsonophagocytic activity (OPA) geometric mean titers (GMTs) were assessed 30 days post-vaccination. Immunoglobulin G (IgG) geometric mean concentrations (GMCs) and geometric mean fold rises for OPA and IgG responses were also evaluated. Solicited systemic and injection site adverse events (AEs) were collected for 5 days post-vaccination, serious AEs were reported throughout study participation, and all other AEs were reported for 30 days post-vaccination.</p><p><strong>Findings: </strong>All three lots of V116 met equivalence criteria based on OPA GMTs for all 21 serotypes. OPA GMTs and IgG GMCs were comparable between the combined V116 lots and PPSV23 for shared serotypes and were higher in the combined V116 lots for serotypes unique to V116. AEs were similar across the three lots of V116 and between the combined V116 lots and PPSV23.</p><p><strong>Conclusions: </strong>V116 exhibited immunogenicity and safety profiles that were consistent across three manufacturing lots.</p><p><strong>Funding: </strong>Funding for this research was provided by Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100748"},"PeriodicalIF":12.8,"publicationDate":"2025-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144340483","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"JAKing down glucocorticoids in giant cell arteritis.","authors":"Tanaz A Kermani, Kenneth J Warrington","doi":"10.1016/j.medj.2025.100744","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100744","url":null,"abstract":"<p><p>Giant cell arteritis is a large-vessel vasculitis affecting the aorta and its branches. While treatment with glucocorticoids is efficacious in the initial management, patients experience relapses and adverse effects. The results of a clinical trial evaluating upadacitinib for treatment of giant cell arteritis have been published.¹ Here, we review the data and the implications for patients with this condition.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":"6 6","pages":"100744"},"PeriodicalIF":12.8,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295068","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Global, multi-center, repeat-dose, phase 2 study of RZ358 (ersodetug), an insulin receptor antibody, for congenital hyperinsulinism.","authors":"Huseyin Demirbilek, Maria Melikyan, Violeta Iotova, Sonya Galcheva, Mehmet Nuri Ozbek, Antonia Dastamani, Nino Kheladze, Kineret Mazor-Aronovitch, Maria Clemente, Susann Empting, Klaus Mohnike, Henrik Thybo Christesen, Paul S Thornton, Diva D De Leon, Davelyn Hood, Erin O'Boyle, Brian K Roberts","doi":"10.1016/j.medj.2025.100611","DOIUrl":"10.1016/j.medj.2025.100611","url":null,"abstract":"<p><strong>Background: </strong>Congenital hyperinsulinism (cHI) is a rare, primarily pediatric disease characterized by dysregulated insulin secretion resulting in severe, persistent hypoglycemia, frequently leading to lifelong neurologic impairments. The safety, pharmacokinetics, and glycemic efficacy of ersodetug, a fully human monoclonal antibody that allosterically and reversibly binds the insulin receptor (INSR) and reduces excess insulin action, are being evaluated for the treatment of cHI-related hypoglycemia.</p><p><strong>Methods: </strong>A global, open-label, phase 2b study (ClinicalTrials.gov: NCT04538989) was conducted in 23 patients with cHI with persistent hypoglycemia on standard-of-care (SOC) therapies. Eligible participants (age ≥2 years) received add-on ersodetug at dose levels between 3 and 9 mg/kg intravenously (i.v.) bi-weekly for 8 weeks in 4 sequential dose cohorts.</p><p><strong>Findings: </strong>Enrolled participants (average age = 6.7 years) on SOC (87% medications; 17% previous pancreatectomy) experienced 13 events/week and 23% time in hypoglycemia at baseline. Ersodetug resulted in predictable, dose-proportional pharmacokinetics. No deaths, adverse drug reactions, study withdrawals, or dose-limiting toxicities occurred. Hypoglycemia (<70 mg/dL) events (self-monitored blood glucose) and time (continuous glucose monitoring) improved from baseline by medians of 59% (p < 0.001) and 54% (p < 0.001), respectively, across pooled dose levels and by 48%-84% (events) and 61%-65% (time) at doses of 6 or 9 mg/kg (p < 0.05) with a nearly universal individual patient response rate. Additional hypoglycemia metrics, including overnight hypoglycemia, similarly improved.</p><p><strong>Conclusion: </strong>Ersodetug was generally well tolerated and significantly improved hypoglycemia in participants with cHI. Ersodetug represents a novel INSR-targeted mechanism of action with the potential to be an effective therapy for all forms of cHI, alone or in combination with other therapies.</p><p><strong>Funding: </strong>Rezolute, Inc. (Redwood City, CA), provided funds.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":" ","pages":"100611"},"PeriodicalIF":12.8,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664610","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"siRNAs targeting ANGPTL3 for mixed dyslipidemia.","authors":"Ioanna Gouni-Berthold","doi":"10.1016/j.medj.2025.100746","DOIUrl":"https://doi.org/10.1016/j.medj.2025.100746","url":null,"abstract":"<p><p>The PROLONG-ANG3 trial demonstrated that two doses of solbinsiran, an siRNA targeting hepatic ANGPTL3, given 90 days apart, decreased concentrations of apoB, triglycerides, LDL-C, non-HDL-C, ANGPTL3, and hepatic fat content at day 180 in adults with mixed dyslipidemia.¹ Long-term studies assessing the safety of solbinsiran and its efficacy in decreasing the risk of atherosclerotic cardiovascular disease are warranted.</p>","PeriodicalId":29964,"journal":{"name":"Med","volume":"6 6","pages":"100746"},"PeriodicalIF":12.8,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295073","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}