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Farnesyltransferase inhibitors: a comprehensive review based on quantitative structural analysis.

Farnesyltransferase inhibitors (FTIs) have mainly been used in cancer therapy. However, more recently, investigations on these inhibitors re … acid and pravastatin for the treatment of Hutchinson-Gilford Progeria syndrome (HGPS) and progeroid laminopathies. Lonafarnib therapy improved cardiovascular status of children with HGPS, by improved peripheral arterial stiffness, bone structure and audiological status in the patients. Other important FTIs such as BMS-214662, LB42908, LB42708, etc are under cli …

Sporadic late onset nemaline myopathy with monoclonal gammopathy of undetermined significance: two cases with long term stability.

Monoclonal gammopathy of undetermined significance (MGUS) associated to sporadic late onset nemaline myopathy (SLONM) is a rare and severely disabling condition of quickly progressive limb girdle acquired myopathy. It is believed by some authors to be due to myotoxicity of light chain deposits. Two female patients were diagnosed with MGUS associated SLONM. In the first case, diagnosis was delayed by 6 years thus giving time for a severe generalized myopathy and cardiomyopathy to develop. A single anti-myeloma chemotherapy with lenalidomide markedly improved and stabilized the patient's conditi

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… In 2006 signs of cardiomyopathy and a respiratory insufficiency appeared, prompting genetic investigations for laminopathies, which were negative. …

PROCEEDINGS OF THE XVII CONGRESS OF THE ITALIAN ASSOCIATION OF MYOLOGY Siracusa, Italy May 31 - June 3, 2017.

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… (Milano) POPDC1 Gene mutations screening in Laminopathies: possible role as a modifier. … The index case of laminopathies Passamano L., D'Ambrosio P., Petillo R., Della Pepa C., De Luca C., Torella A., Papa A.A., Palladino A., Novelli A., Nigro V., Politano L. …

Global first-in-class drugs approved in 2023-2024: Breakthroughs and insights.

First-in-class (FIC) drugs are considered the main drivers of new drug discovery and have novel targets and mechanisms. The proportion of FIC drugs among new drugs provides fundamental guidance for both academic and pharmaceutical research. From a global perspective, 81 FIC drugs were approved in 2023 and 2024. Among them, small-molecule drugs account for a greater percentage (51.9%), illustrating the discovery of new chemical entities. Macromolecule drugs (48.1%), which mainly consist of antibody analogs, represent a growing trend as new biotechnology techniques have emerged. In terms of FIC

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… maribavir small molecule post-transplant cytomegalovirus (CMV) infection human CMV pUL97 viral protein kinase inhibitor 28 berdazimer small molecule molluscum contagiosum mechanism of action unknown 29 lonafarnib small molecule Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies … molecule | post-transplant cytomegalovirus (CMV) infection | human CMV pUL97 viral protein kinase inhibitor 28 | berdazimer | small molecule | molluscum contagiosum | mechanism of action unknown 29 | lonafarnib | small molecule | Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies …

Резюме 18th Неевромускульная научно-исследовательская конференция Соединенного Королевства 15th и 16th апрель 2025.

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Alzheimer's Disease: Cellular and Pharmacological Aspects.

Alzheimer's disease was described more than 100 years ago and despite the fact that several molecules are being tested for its treatment, which are in phase III trials, the disease continues to progress. The main problem is that these molecules function properly in healthy neurons, while neuronal pathology includes plasma membrane disruption, malfunction of various organelles, and hyperphosphorylation of Tau and amyloid plaques. The main objective of this article is the discussion of a neuronal restoration therapy, where molecules designed for the treatment of Alzheimer's disease would probabl

Cardiomyopathy: pathogenesis and therapeutic interventions.

Cardiomyopathy is a group of disease characterized by structural and functional damage to the myocardium. The etiologies of cardiomyopathies are diverse, spanning from genetic mutations impacting fundamental myocardial functions to systemic disorders that result in widespread cardiac damage. Many specific gene mutations cause primary cardiomyopathy. Environmental factors and metabolic disorders may also lead to the occurrence of cardiomyopathy. This review provides an in-depth analysis of the current understanding of the pathogenesis of various cardiomyopathies, highlighting the molecular and

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… They also proposed that PDGF receptor beta (PDGFRB) might represent a promising therapeutic target for this condition. 326 Chen et al. identified the E2F/DNA damage response/TP53 axis as a mechanism underlying the development of DCM in laminopathies and suggested it as a potential target for interventions …

Unlocking the therapeutic potential of cellular mechanobiology.

Mechanobiology is a rapidly advancing field at the intersection of biology, physics, and engineering that reveals how mechanical forces shape cellular behavior, tissue architecture, and disease progression. By elucidating how cells sense and transduce mechanical cues, mechanobiology has fundamentally advanced our understanding of processes ranging from migration and differentiation to immune responses and tissue remodeling. These advances have driven the development of innovative biophysical tools and engineered biomaterials that enable precise modulation of the cellular microenvironment. Tran

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… It has revealed how nuclear deformability is altered in diseases such as laminopathies, with implications for cell migration and differentiation ( 122 ). High-throughput microfluidic micropipette assays have also identified how force duration influences spheroid deformability ( 126 ). …

Targeting Cellular Senescence in Aging and Age-Related Diseases: Challenges, Considerations, and the Emerging Role of Senolytic and Senomorphic Therapies.

Cellular senescence is characterized by the permanent arrest of cell proliferation and is a response to endogenous and exogenous stress. The continuous accumulation of senescent cells (SnCs) in the body leads to the development of aging and age-related diseases (such as neurodegenerative diseases, cancer, metabolic diseases, cardiovascular diseases, and osteoarthritis). In the face of the growing challenge of aging and age-related diseases, several compounds have received widespread attention for their potential to target SnCs. As a result, senolytics (compounds that selectively eliminate SnCs

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… Hutchinson-Gilford progeria syndrome (HGPS), which is based on mutations in the LMNA gene and the gene encoding the interacting protein, is one of the most serious diseases in laminopathies [ 188 ]. LMNA encodes two major alternate splicing transcripts, producing laminin A and laminin C [ 188 ]. …

Tumor biomarkers for diagnosis, prognosis and targeted therapy.

Tumor biomarkers, the substances which are produced by tumors or the body's responses to tumors during tumorigenesis and progression, have been demonstrated to possess critical and encouraging value in screening and early diagnosis, prognosis prediction, recurrence detection, and therapeutic efficacy monitoring of cancers. Over the past decades, continuous progress has been made in exploring and discovering novel, sensitive, specific, and accurate tumor biomarkers, which has significantly promoted personalized medicine and improved the outcomes of cancer patients, especially advances in molecu

Integumentary System

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Резюме

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Ход работы сессии 28th Европейский конгресс педиатрической ремуматологии (ПСР) 2022) Прага, Чешская Республика. 20-23 сентябрь 2022.

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