Tamoxifen may contribute to preserve cardiac function in Duchenne muscular dystrophy DOI Creative Commons

Bettina Henzi,

Sebastiano A. G. Lava,

Carlos Spagnuolo

et al.

European Journal of Pediatrics, Journal Year: 2024, Volume and Issue: 183(9), P. 4057 - 4062

Published: July 3, 2024

Abstract Duchenne muscular dystrophy is life-limiting. Cardiomyopathy, which mostly ensues in the second decade of life, main cause death. Treatment options are still limited. The TAMDMD (NCT03354039) trial assessed motor function, muscle strength and structure, laboratory biomarkers, safety 79 ambulant boys with genetically confirmed dystrophy, 6.5–12 years age, receiving either daily tamoxifen 20 mg or placebo for 48 weeks. In this post-hoc analysis, available echocardiographic data patients recruited at one study centre were retrieved compared before after treatment. Data from 14 patients, median 11 (interquartile range, IQR, 11–12) age was available. Baseline demographic characteristics similar participants assigned to ( n = 7) 7). Left ventricular end-diastolic diameter group (median IQR) 39 (38–41) mm baseline 43 (38–44) end, while it 44 (41–46) 41 (37–46) treatment group. fractional shortening 35% (32–38%) 33% (32–36%) treatment, 34% (33–34%) (33–35%) end. No signals detected. Conclusion : This hypothesis-generating analysis suggests that over weeks well tolerated may help preserving cardiac structure function dystrophy. Further studies justified. ClinicalTrials.gov Identifier EudraCT 2017–004554–42, NCT03354039 What known: • (DMD) Cardiomyopathy life Tamoxifen reduced fibrosis mice improved cardiomyocyte human-induced pluripotent stem cell-derived cardiomyocytes. new: among boys, treated weeks, well-tolerated. A visual trend left-ventricular dimensions better systolic preservation generates hypothesis a potential beneficial effect DMD cardiomyopathy.

Language: Английский

Repurposing Drugs for the Treatment of COVID-19 and Its Cardiovascular Manifestations DOI Creative Commons
Rui‐Sheng Wang, Joseph Loscalzo

Circulation Research, Journal Year: 2023, Volume and Issue: 132(10), P. 1374 - 1386

Published: May 11, 2023

COVID-19 is an infectious disease caused by SARS-CoV-2 leading to the ongoing global pandemic. Infected patients developed a range of respiratory symptoms, including failure, as well other extrapulmonary complications. Multiple comorbidities, hypertension, diabetes, cardiovascular diseases, and chronic kidney are associated with severity increased mortality COVID-19. infection also causes complications, myocarditis, myocardial injury, heart arrhythmias, acute coronary syndrome, venous thromboembolism. Although variety methods have been many clinical trials launched for drug repositioning COVID-19, treatments that consider manifestations comorbidities specifically limited. In this review, we summarize recent advances in experimental repositioning, high-throughput screening, omics data-based, network medicine-based computational particular attention on those We discuss prospective opportunities potential repurposing drugs treat complications

Language: Английский

Citations

17

Targeting mitochondrial impairment for the treatment of cardiovascular diseases: From hypertension to ischemia-reperfusion injury, searching for new pharmacological targets DOI
Simona Todisco, Biagia Musio, Vito Pesce

et al.

Biochemical Pharmacology, Journal Year: 2023, Volume and Issue: 208, P. 115405 - 115405

Published: Jan. 2, 2023

Language: Английский

Citations

14

Bioinformatic platforms for clinical stratification of natural history of atherosclerotic cardiovascular diseases DOI
Giuditta Benincasa, Rosa Suades, Teresa Padró

et al.

European Heart Journal - Cardiovascular Pharmacotherapy, Journal Year: 2023, Volume and Issue: 9(8), P. 758 - 769

Published: Aug. 10, 2023

Abstract Although bioinformatic methods gained a lot of attention in the latest years, their use real-world studies for primary and secondary prevention atherosclerotic cardiovascular diseases (ASCVD) is still lacking. Bioinformatic resources have been applied to thousands individuals from Framingham Heart Study as well health care-associated biobanks such UK Biobank, Million Veteran Program, CARDIoGRAMplusC4D Consortium randomized controlled trials (i.e. ODYSSEY, FOURIER, ASPREE, PREDIMED). These contributed development polygenic risk scores (PRS), which emerged novel potent genetic-oriented tools, able calculate individual ASCVD predict response therapies statins proprotein convertase subtilisin/kexin type 9 inhibitor. are first cause death around world including coronary heart disease (CHD), peripheral artery disease, stroke. To achieve goal precision medicine personalized therapy, advanced platforms set link clinically useful indices heterogeneous molecular data, mainly epigenomics, transcriptomics, metabolomics, proteomics. The DIANA study found that differential methylation ABCA1, TCF7, PDGFA, PRKCZ significantly discriminated patients with acute syndrome healthy subjects expression levels positively associated CK-MB serum concentrations. ARIC revealed several plasma proteins, acting or not lipid metabolism, potential role determining different pleiotropic effects each subject. implementation high-throughput techniques into traditional prediction emerging more accurate practice stratify earlier life favour timely tailored reduction strategies. Of note, radiogenomics aims combine imaging features extracted instance by computed tomography angiography biomarkers create CHD diagnostic algorithms characterize lesions myocardial abnormalities. current view could be clinical value prevention, stratification, treatment ASCVD.

Language: Английский

Citations

14

Revolutionizing Neurological Disorder Treatment: Integrating Innovations in Pharmaceutical Interventions and Advanced Therapeutic Technologies DOI

Rimpi Arora,

Ashish Baldi

Current Pharmaceutical Design, Journal Year: 2024, Volume and Issue: 30(19), P. 1459 - 1471

Published: April 15, 2024

Neurological disorders impose a significant burden on individuals, leading to disabilities and reduced quality of life. However, recent years have witnessed remarkable advancements in pharmaceutical interventions aimed at treating these disorders. This review article aims provide an overview the latest innovations breakthroughs neurological disorder treatment, with specific focus key therapeutic areas such as Alzheimer's disease, Parkinson's multiple sclerosis, epilepsy, stroke. explores emerging trends drug development, including identification novel targets, development innovative delivery systems, application personalized medicine approaches. Furthermore, it highlights integration advanced technologies gene therapy, optogenetics, neurostimulation techniques. These hold promise for precise modulation neural circuits, restoration neuronal function, even disease modification. While offer hopeful prospects more effective tailored treatments, challenges need improved diagnostic tools, new targets intervention, optimization methods will remain. By addressing continuing invest research collaboration, we can revolutionize treatment significantly enhance lives those affected by conditions.

Language: Английский

Citations

6

Innovative cancer therapy: Unleashing the potential of macromolecule-loaded mesoporous bioactive glasses for precision diagnosis and treatment DOI
Ali Mohammad Amani, Lobat Tayebi, Ehsan Vafa

et al.

International Journal of Pharmaceutics, Journal Year: 2024, Volume and Issue: unknown, P. 124847 - 124847

Published: Oct. 1, 2024

Language: Английский

Citations

6

Single-Cell Meta-Analysis of Neutrophil Activation in Kawasaki Disease and Multisystem Inflammatory Syndrome in Children Reveals Potential Shared Immunological Drivers DOI Creative Commons
Jan Vincent B. Beltran,

Fang‐Ping Lin,

Chaw‐Liang Chang

et al.

Circulation, Journal Year: 2023, Volume and Issue: 148(22), P. 1778 - 1796

Published: Oct. 31, 2023

Kawasaki disease (KD) and multisystem inflammatory syndrome in children (MIS-C) share similar clinical manifestations, including cardiovascular complications, suggesting underlying immunopathogenic processes. Aberrant neutrophil activation may play a crucial role the shared pathologies of KD MIS-C; however, associated pathogenic mechanisms molecular drivers remain unknown.

Language: Английский

Citations

13

Three-dimensional cardiac models: a pre-clinical testing platform DOI Creative Commons
Eline Groen, Christine L. Mummery, Loukia Yiangou

et al.

Biochemical Society Transactions, Journal Year: 2024, Volume and Issue: 52(3), P. 1045 - 1059

Published: May 23, 2024

Major advancements in human pluripotent stem cell (hPSC) technology over recent years have yielded valuable tools for cardiovascular research. Multi-cell type 3-dimensional (3D) cardiac models particular, are providing complementary approaches to animal studies that better representatives than simple 2-dimensional (2D) cultures of differentiated hPSCs. These 3D can be broadly divided into two categories; namely those generated through aggregating pre-differentiated cells and form self-organizing structures during their vitro differentiation from either replicate aspects development or enable the examination interactions among constituent types, with some these showing increased maturity compared 2D systems. Both groups already emerged as physiologically relevant pre-clinical platforms studying heart disease mechanisms, exhibiting key functional attributes heart. In this review, we describe different organoid derived hPSCs, generation methods, applications research use drug screening. We also address current limitations challenges testing propose potential improvements enhance efficacy discovery.

Language: Английский

Citations

4

MEK inhibitors: a promising targeted therapy for cardiovascular disease DOI Creative Commons

Khaled Abdelsattar Kassem Mohammed,

Paolo Madeddu, Elisa Avolio

et al.

Frontiers in Cardiovascular Medicine, Journal Year: 2024, Volume and Issue: 11

Published: July 1, 2024

Cardiovascular disease (CVD) represents the leading cause of mortality and disability all over world. Identifying new targeted therapeutic approaches has become a priority biomedical research to improve patient outcomes quality life. The RAS-RAF-MEK (mitogen-activated protein kinase kinase)-ERK (extracellular signal-regulated kinase) pathway is gaining growing interest as potential signaling cascade implicated in pathogenesis CVD. This pivotal regulating cellular processes like proliferation, growth, migration, differentiation, survival, which are vital maintaining cardiovascular homeostasis. In addition, ERK involved controlling angiogenesis, vascular tone, myocardial contractility, oxidative stress. Dysregulation this been linked cell dysfunction cardiac pathological remodeling, contribute onset progression Recent ongoing provided insights into interventions targeting RAS-RAF-MEK-ERK pathologies. Preclinical studies have demonstrated efficacy therapy with MEK inhibitors (MEKI) attenuating activation mitigating CVD animal models. article, we first describe how contributes preserving health. We then summarize current knowledge roles played by development disorders, including atherosclerosis, infarction, hypertrophy, heart failure, aortic aneurysm. finally report novel strategies for these CVDs encompassing MEKI discuss advantages, challenges, future developments therapeutics.

Language: Английский

Citations

4

Microfluidics for diagnosis and treatment of cardiovascular disease DOI

Yonggeng Ma,

Chenbin Liu, Siyu Cao

et al.

Journal of Materials Chemistry B, Journal Year: 2022, Volume and Issue: 11(3), P. 546 - 559

Published: Dec. 5, 2022

Cardiovascular disease (CVD), a type of circulatory system related to the lesions cardiovascular system, has become one main diseases that endanger human health. Currently, clinical diagnosis most CVDs relies on combination imaging technology and blood biochemical test. However, existing technologies for still have limitations in terms specificity, detection range, cost. In order break through current bottleneck, microfluidic with advantages low cost, simple instruments easy integration, been developed play an important role early prevention, treatment CVDs. Here, we reviewed recent various applications CVDs, including devices detecting CVD markers, models based microfluidic, used drug screening delivery. addition, briefly looked forward prospects challenges microfluidics

Language: Английский

Citations

17

Drug Repurposing and Nanotechnology for Topical Skin Cancer Treatment: Redirecting toward Targeted and Synergistic Antitumor Effects DOI Creative Commons
Maria Auxiliadora Parreiras Martins, Francisco Veiga, Ana Cláudia Paiva‐Santos

et al.

ACS Pharmacology & Translational Science, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 23, 2025

Skin cancer represents a major health concern due to its rising incidence and limited treatment options. Current treatments (surgery, chemotherapy, radiotherapy, immunotherapy, targeted therapy) often entail high costs, patient inconvenience, significant adverse effects, therapeutic efficacy. The search for novel options is also marked by the capital investment extensive development involved in drug discovery process. In response these challenges, repurposing existing drugs topical application optimizing their delivery through nanotechnology could be answer. This innovative strategy aims combine advantages of known pharmacological background commonly used expedite development, with nanosystem-based formulations, which among other allow improved skin permeation retention overall higher efficacy safety. present review provides critical analysis repurposed such as doxycycline, itraconazole, niclosamide, simvastatin, leflunomide, metformin, celecoxib, formulated into different nanosystems, namely, nanoemulsions nanoemulgels, nanodispersions, solid lipid nanoparticles, nanostructured carriers, polymeric hybrid lipid-polymer electrospun nanofibrous scaffolds, liposomes liposomal gels, ethosomes ethosomal aspasomes, outcomes battle against cancer. Enhanced antitumor effects on melanoma nonmelanoma research models are highlighted, some nanoparticles even showing intrinsic anticancer properties, leading synergistic effects. explored findings highly evidence potential approaches complement currently available strategies hope that might one day reach pharmaceutical market.

Language: Английский

Citations

0