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Hypophosphatasia: Results of a Country-Wide Selective Screening Program Using NGS Technology as a First-Tier Test.

Hypophosphatasia (HPP) is a rare hereditary metabolic disorder caused by nucleotide variants (NVs) in the ALPL gene, leading to a deficiency of tissue-nonspecific alkaline phosphatase (TNSALP). HPP has a variable clinical presentation (such as impaired mineralization of bones and teeth, respiratory dysfunctions, muscle weakness, muscle and bone pain, growth deficiency, nephrocalcinosis, seizures, encephalopathy, intracranial hypertension, and intellectual disability) overlapping with other disorders which often delays a correct diagnosis. In this study, we present the results of a two-center (

Эффективность и безопасность терапии клеток CAR-T и биконкретных антител в повторной/отредактированной миеломе с повреждением ренальности: анализ пропенсиентности.

Иммунотерапия с перенаправкой Т, включая химериковую антигенную рецепторную терапию T-клеток (CAR-T) и биконкретные антитела (BSAbs), изменила лечение повторной/охлажденной миеломы (RRMMM). В то же время в ходе важнейших регистрационных испытаний, как правило, исключались пациенты с существенными почечными нарушениями (РИ), что создавало критический разрыв в данных о населении, в котором болезни почек 20-50% пациентов с диагнозом. Прямого сопоставления результатов по расчетному спектру широтной фильтрации (eGFR) для обоих вариантов не проводится.МЕТОДЫ: Мы провели ретроспективное когортное исс

Global perspectives on lupus nephritis: a review of clinical trials and therapeutic innovations.

BACKGROUND AND OBJECTIVES: Lupus nephritis (LN) remains a leading cause of morbidity and mortality in systemic lupus erythematosus (SLE). Although recent years have witnessed the approval of several targeted therapies, achieving long-term, drug-free remission remains challenging. This commentary evaluates the global landscape of interventional LN clinical trials from 2001 to 2026 to identify emerging trends and strategic gaps in drug development. METHODS: We analyzed 200 interventional pharmacological trials retrieved from three major global registries: ClinicalTrials.gov, Chinadrugtrials.org

Disturbing the Redox Balance Using Buthionine Sulfoximine Radiosensitized Somatostatin Receptor-2 Expressing Pre-Clinical Models to Peptide Receptor Radionuclide Therapy with 177Lu-DOTATATE.

Peptide receptor radionuclide therapy with 177Lu-DOTATATE improves the outcome of patients with somatostatin receptor (SSTR)-expressing neuroendocrine tumours. Nevertheless, stable disease has been the main response pattern observed, with some rare complete responses. Lu-177 exerts about two-thirds of its biological effects via the indirect effects of ionizing radiation that generate reactive oxygen species, eventually leading to oxidative damage and cell death. This provides a rationale for targeting the antioxidant defence system in combination with 177Lu-DOTATATE. In the present study, the

Mechanism-based prediction of drug synergy via network controllability analysis of therapeutic pathways in intractable diseases.

Identifying effective drug combinations for multidrug therapies in intractable diseases remains challenging, and drug synergy depends on regulatory mechanisms. Herein, we introduce a mechanistic framework that redefines drug combination strategies into two distinct synergy paradigms-"boost" and "complement"-and present a network-based method called SYNERGIE to predict associated combinations across diseases. For diseases lacking known therapeutic targets, we introduced network controllability analysis to estimate therapeutic pathways and target molecules. SYNERGIE integrates drug-disease and d

Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies.

The paradigm of pediatric drug development has been evolving in a "carrot-and-stick"-based tactic to address population-specific issues. However, the off-label prescription of adult medicines to pediatric patients remains a feature of clinical practice, which may compromise the age-appropriate evaluation of treatments. Therefore, the United States and the European Pediatric Formulation Initiative have recommended applying nanotechnology-based delivery systems to tackle some of these challenges, particularly applying inorganic, polymeric, and lipid-based nanoparticles. Connected with these, adv

Overview of Ankle Arthropathy in Hereditary Hemochromatosis.

Hereditary hemochromatosis (HH) is an autosomal recessive bleeding disorder characterized by tissue overload of iron. Clinical systemic manifestations in HH include liver disease, cardiomyopathy, skin pigmentation, diabetes mellitus, erectile dysfunction, hypothyroidism, and arthropathy. Arthropathy with joint pain is frequently reported at diagnosis and mainly involves the metacarpophalangeal and ankle joints, and more rarely, the hip and knee. Symptoms in ankle joints are in most cases non-specific, and they can range from pain and swelling of the ankle to deformities and joint destruction.

Талидомид взаимодействует с воспалением в идиопатическом фиброзе легких.

" Трагедия Талидомида " является важной вехой в истории фармацевтической промышленности. Несмотря на ограниченные клинические испытания, прилагаются постоянные усилия по изучению Талидомида в качестве лекарства от рака и подстрекательских заболеваний, таких, как ревматуидный артрит, проказа, и COVID-19. В рамках этого обзора основное внимание уделяется возможностям борьбы с воспалением путем повторного использования талидомида для лечения идиопатического фиброза легких (ИПФ). Статьи были найдены в базе данных Scopus, сортированы и рассмотрены отдельные статьи. Содержание включает в с

Vitamin D: A Bridge between Kidney and Heart.

Chronic kidney disease (CKD) and cardiovascular disease (CVD) are highly prevalent conditions, each significantly contributing to the global burden of morbidity and mortality. CVD and CKD share a great number of common risk factors, such as hypertension, diabetes, obesity, and smoking, among others. Their relationship extends beyond these factors, encompassing intricate interplay between the two systems. Within this complex network of pathophysiological processes, vitamin D has emerged as a potential linchpin, exerting influence over diverse physiological pathways implicated in both CKD and CV

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… Beyond that, vitamin D is involved in calcium homeostasis by increasing renal reabsorption, increasing calcium release from bone by osteoclasts, and stimulating the production of calcium transporters [ 132 ]. …

The potential of dibenzazepine carboxamides in cancer therapy.

Cancer is a leading cause of mortality worldwide, with most conventional treatments lacking efficacy and having significant challenges like drug resistance. Finding new molecules is quite challenging in terms of cost, time and setbacks. Hence, drug repurposing is considered sensible for skipping the long process of drug development. Dibenzazepine carboxamides, as traditional anticonvulsants, primarily function by blocking voltage-gated sodium channels, which not only mitigate seizures but also influence mood disorders through modulation of serotonin and dopamine. Recent studies have uncovered

Acute kidney injury: pathogenesis and therapeutic interventions.

Acute kidney injury (AKI) is a prevalent clinical condition that is associated with unacceptably high morbidity and mortality, as well as the development of chronic kidney disease (CKD). The pathogenesis of AKI is highly complex and heterogeneous, primarily attributed to metabolic disturbances arising from the disease itself and the administration of medications related to treatment. In recent years, AKI in cancer patients is highly concerned. The emergence of AKI caused injuries and dysfunction of remote organs but also enhanced the health-care costs. It's essential for early recognition of A

Development of Targeted Alpha Particle Therapy for Solid Tumors.

Targeted alpha-particle therapy (TAT) aims to selectively deliver radionuclides emitting α-particles (cytotoxic payload) to tumors by chelation to monoclonal antibodies, peptides or small molecules that recognize tumor-associated antigens or cell-surface receptors. Because of the high linear energy transfer (LET) and short range of alpha (α) particles in tissue, cancer cells can be significantly damaged while causing minimal toxicity to surrounding healthy cells. Recent clinical studies have demonstrated the remarkable efficacy of TAT in the treatment of metastatic, castration-resistant prosta

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… Other important research regarding linkers has uncovered different methods to reduce kidney toxicity, via the renal reabsorption of radiolabeled peptides and antibody fragments, as they are filtered by the glomerulus. …

Selected abstracts from the XXXVIII National Congress of the Italian Society of Hypertension (SIIA)

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Emergency Care

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Экстракорпоральные стратегии свободного освещения и гемоглозы в эпоху эффективной антимиеломной терапии.

Повреждение реналов затрагивает до половины пациентов с недавно поставленным диагнозом множественной миеломы, и примерно 2-4% из них требуют диализа при презентации, чаще всего из-за нефропатии легкой цепи. У этих пациентов восстановление почек связано со скоростью и величиной сокращения свободных световых цепей (SFLC) в наблюдательных группах - ассоциации, которая в основном отражает гематологическую реакцию на химиотерапию; постепенное преимущество удаления циркулирующих SFLC с помощью экстракорпоральных средств остается неопределенным, и эта повествование построена вокруг этого вопроса в ны

Heart failure with preserved ejection fraction in cancer patients and survivors. A scientific statement of the Heart Failure Association of the ESC and the ESC Council of Cardio-Oncology.

Heart failure with preserved ejection fraction (HFpEF) is increasingly recognized in cancer patients and survivors, yet it remains underdiagnosed and its epidemiology largely unknown. This statement underscores the imperative need to include HFpEF in the cardiotoxicities identified during or after anticancer treatments. It also highlights the prognostic value of pre-existing HFpEF for periprocedural and cardiotoxicity risk and it discusses the challenges in the diagnosis and treatment of HFpEF in cancer patients. It also explores the aetiologic role of anticancer therapies (chemotherapy, targe

Approaches and Challenges in the Management of Multiple Myeloma in the Very Old: Future Treatment Prospects.

The increasing incidence of geriatric patients with multiple myeloma has elevated concerns in clinical practice. While the introduction of novel therapeutic agents has substantially improved outcomes in younger patients with myeloma, poorer outcomes remain in older patients. Managing older patients requires a multidisciplinary team approach to consider factors that may influence both treatment selection and outcomes. Aging is associated with remodeling of vital organs, physiological downregulations of basal metabolism, susceptibility to multiple comorbidities with ultimate frailty, thereby con

Radiolabeled Peptides and Antibodies in Medicine.

Radiolabeled peptides are a relatively new, very specific radiotracer group, which is still expanding. This group is very diverse in terms of peptide size. It contains very small structures containing several amino acids and whole antibodies. Moreover, radiolabeled peptides are diverse in terms of the binding aim and therapeutic or diagnostic applications. The majority of this class of radiotracers is utilized in oncology, where the same structure can be used in therapy and diagnostic imaging by varying the radionuclide. In this study, we collected new reports of radiolabeled peptide applicati

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… In conclusion, Tolmachev et al. suggested avoiding the combination of radiocopper and NOTA/NODAGA amido derivatives with proteins displaying high renal reabsorption. 114 Sandberg et al. investigated a novel affibody, ABY-025, conjugated with 68 Ga and 111 In. …

Anti-Infective-Associated AKI: A Narrative Review of the Epidemiology, Mechanisms, Risk Factors, Biomarkers, Clinical Course, Monitoring, Prevention, and Therapeutic Strategies.

Acute kidney injury (AKI) occurs commonly in hospitalized patients, especially patients in intensive care units (ICUs). Medications are among the major causative factors of AKI. This narrative review addressed and updated different aspects of anti-infective-associated AKI, including amphotericin B, cidofovir, foscarnet, polymyxins, vancomycin, and aminoglycosides. There is no standard definition or operational criteria to describe anti-infective-associated AKI. Characteristically, it usually occurs during the first two weeks of treatment and is typically dose dependent. Functional resolution o

Multidisciplinary Approach to Spinal Metastases and Metastatic Spinal Cord Compression-A New Integrative Flowchart for Patient Management.

Metastatic spine disease (MSD) and metastatic spinal cord compression (MSCC) are major causes of permanent neurological damage and long-term disability for cancer patients. The development of MSD is pathophysiologically framed by a cooperative interaction between general mechanisms of bone growth and specific mechanisms of spinal metastases (SM) expansion. SM most commonly affects the thoracic spine, even though multiple segments may be affected concomitantly. The great majority of SM are extradural, while intradural-extramedullary and intramedullary metastases are less frequently seen. The ma