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Abstracts of the 19th International Congress on Neuromuscular Diseases 7th – 11th July 2026

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… Eugenio Mercuri 3 1 University of Florida, Gainesville, Florida, United States. 2 Italfarmaco S.p.A., Milan, Italy. 3 Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy Background: The phase 3 EPIDYS trial ( NCT02851797 ) was a … James F Howard Jr 12 1 Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy. 2 University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom. 3 University of South Florida Morsani College of Medicine, Tampa, FL, United States. 4 University of California Irvine, Orange, …

Rapid Fire Presentations

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Biology and Diseases of Dogs

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Абстрактный сборник ISEV2022.

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The Clinical Relevance of Selected Cytokines in Newly Diagnosed Multiple Myeloma Patients.

Multiple myeloma (MM) is the second most common hematological neoplasm. Cytokines, chemokines, and their receptors, induced by the microenvironment of MM, participate in tumor growth, the attraction of leukocytes, cell homing, and bone destruction. This study aimed to assess the correlation between the pretreatment serum concentrations of interleukin-6 (IL-6), interleukin-8 (IL-8), angiogenic chemokine monocyte chemoattractant protein-1 (MCP-1), and vascular endothelial growth factor (VEGF) and the clinical outcomes and survival of patients newly diagnosed with MM. The study group consisted of

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… Di Raimondo F., Azzaro M.P., Palumbo G., Bagnato S., Giustolisi G., Florida P.M., Sortino G., Giustolisi R. Angiogenic factors in multiple myeloma: Higher levels in bone marrow than in peripheral blood. Haematologica. 2000;85:800–805. 84. …

Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor (rhu GM-CSF) as Adjuvant Therapy for Invasive Fungal Diseases.

BackgroundSargramostim (yeast-derived, glycosylated recombinant human granulocyte-macrophage colony-stimulating factor [rhu GM-CSF]) augments innate and adaptive immune responses and accelerates hematopoietic recovery of chemotherapy-induced neutropenia. However, considerably less is known about its efficacy as adjunctive immunotherapy against invasive fungal diseases (IFDs).MethodsThe clinical courses of 15 patients with pediatric malignancies and IFDs treated adjunctively with sargramostim at a single institution were analyzed in a retrospective cohort review. Further, a systematic review of

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… Haque 2019 (Florida) [ 38 ] | 49 yo M | Liver transplant | Surgical site mucormycosis ( Rhizopus ) | No | Yes (prior fluconazole) | Rhu GM-CSF x11 d (dose NR) | Amphotericin B, micafungin, posaconazole | No effect on infection; subsequent worsening hypoxia, metabolic acidosis, with bradycardic arrest …

Unlocking the potential: advancements and applications of gene therapy in severe disorders.

INTRODUCTION: Several severe disorders, such as inherited diseases (e.g. cystic fibrosis and beta thalassemia), genetic diseases (e.g. malignant tumors and diabetes), and infectious diseases (e.g. HIV) are pose significant challenges to human health. BACKGROUND: Over the past few decades, researchers have been working on gene therapies, and currently, terrible dreams have come true. To date, the Food and Drug Administration (FDA) has approved multiple gene therapies such as Kynamro for familial hypercholesterolaemia, Exondys51 for duchenne muscular dystrophy, Spinraza for spinal muscular atro

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… Huntington’s disease | NCT06444217 | In-Vitro | Trans-splicing | NA | University Hospital, Angers Pompe’s disease | NCT00976352 | Intramuscular injection | rAAV1-CMV-hGAA | II | University of Florida Parkinson’s disease | NCT02418598 | Intraputaminal injection | AAV-hAADC-2 | II | Jichi Medical University …

Оптимизированная внутриназальная доставка сегестерона ацетатата в мозг с использованием наноэмульсии и микроэмульсии.

Segesteron aцетат (SA) является селективным и мощным агонистом рецептора прогестерона с потенциальным применением в лечении неврологических заболеваний, таких, как множественный склероз и инсульт. В этом исследовании микроэмульсия (МЭ) и three Наноэмульсии (NEs) с целевой концентрацией SA 0.48 Мг/г были разработаны для интраназального администрирования и подробно описаны в отношении их физико-химических свойств, устойчивости, антимикробной активности и безопасности (на первичных кортикальных клетках, тесте на яйцеклетки Hen/s, модели MucilAirTM и на крысах). Измерялись уровни плазмы и SA мозга

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… Transepithelial electrical resistance For tissue resistance measurements, an Epithelial Volt/Ohm Meter (EVOM™) from World Precision Instruments (Sarasota, Florida, USA) was used. KRB was placed in a Petri dish and incubated at 37 °C with 5% CO 2 for approximately 15 min to reach equilibrium. …

Exercise as a Therapeutic Strategy to Improve Cerebrovascular Function and Cognition in Breast Cancer Survivors: A Scoping Review.

Breast cancer is the most diagnosed cancer globally. While the breast cancer prevalence continues to rise, so too do patient survival rates, thus resulting in a large survivor population. Up to 75% of this population report experiencing cancer-related cognitive impairment during their cancer journey, thus reducing their quality of survivorship. This review systematically evaluates the effect of physical activity and exercise training on cerebrovascular function and cognition in breast cancer survivors. Cross-sectional, intervention or observational studies that examined the effect of acute or

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… McLaughlin M., Florida-James G., Ross M. Breast cancer chemotherapy vascular toxicity: A review of mediating mechanisms and exercise as a potential therapeutic. Vasc. Biol. 2021;3:R106–R120. doi: 10.1530/VB-21-0013. 67. Bagnall-Moreau C., Chaudhry S., Salas-Ramirez K., Ahles T., Hubbard K. …

Current Trends and Challenges in the Clinical Translation of Nanoparticulate Nanomedicines: Pathways for Translational Development and Commercialization.

The use of nanotechnology in medicine has the potential to have a major impact on human health for the prevention, diagnosis, and treatment of diseases. One particular aspect of the nanomedicine field which has received a great deal of attention is the design and development of nanoparticulate nanomedicines (NNMs) for drug delivery (i.e., drug-containing nanoparticles). NNMs are intended to deliver drugs via various mechanisms: solubilization, passive targeting, active targeting, and triggered release. The NNM approach aims to increase therapeutic efficacy, decrease the therapeutically effecti

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… Tolerability of liposomes in vivo , in Liposome Technology. 3 , ed Gregoriadis G. ( Florida : CRC Press ), 345 – 83 . Storm G. ten Kate M. T. Working P. K. Bakker-Woudenberg I. A. ( 1998 ). …

Burden of cancer in the Eastern Mediterranean Region, 2005-2015: findings from the Global Burden of Disease 2015 Study.

ObjectivesTo estimate incidence, mortality, and disability-adjusted life years (DALYs) caused by cancer in the Eastern Mediterranean Region (EMR) between 2005 and 2015.MethodsVital registration system and cancer registry data from the EMR region were analyzed for 29 cancer groups in 22 EMR countries using the Global Burden of Disease Study 2015 methodology.ResultsIn 2015, cancer was responsible for 9.4% of all deaths and 5.1% of all DALYs. It accounted for 722,646 new cases, 379,093 deaths, and 11.7 million DALYs. Between 2005 and 2015, incident cases increased by 46%, deaths by 33%, and DALYs

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… Peter Memiah, PhD, University of West Florida, Pensacola, FL, United States. Ziad A. Memish, MD, Saudi Ministry of Health, Riyadh, Saudi Arabia; College of Medicine, Alfaisal University, Riyadh, Saudi Arabia. Walter Mendoza, MD, United Nations Population Fund, Lima, Peru. Tuomo J. …

Global Panoramic analysis of clinical research in cell therapy: clinical trial landscape, marketed products, and regulatory trends.

IntroductionCell therapy is a vital field in modern medicine. This review assesses its global clinical development landscape, approved products, and regional regulatory characteristics.MethodsA comprehensive search and analysis of global clinical trial databases (e.g., https://clinicaltrials.gov/ClinicalTrials.gov), regulatory agency announcements, and relevant literature up to October 2025 was conducted. Key data points regarding trial numbers, phases, therapy types, and approved products were extracted and analyzed descriptively by region.ResultsA cumulative total of 10,373 cell therapy clin

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… Footnotes Edited by: Ricardo Ferraz , Polytechnic Institute of porto, Portugal Reviewed by: Alejandrina Hernandez-Lopez , Autonomous University of the State of Morelos, Mexico Chiranjeevi Sainatham , University of Florida, United States Xiaoyuan Chen , Tsinghua University, China Data availability …

Recent advances in management of COVID-19: A review.

The coronavirus disease 2019 (COVID-19) pandemic caused and is still causing significant mortality and economic consequences all over the globe. As of today, there are three U.S Food and Drug administration (FDA) approved vaccines, Pfizer-BioNTech, Moderna and Janssen COVID-19 vaccine. Also, the antiviral drug remdesivir and two combinations of monoclonal antibodies are authorized for Emergency use (EUA) in certain patients. Furthermore, baricitinib was approved in Japan (April 23, 2021). Despite available vaccines and EUA, pharmacological therapy for the prevention and treatment of COVID-19 i

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… In clinical settings, a retrospective cohort study including ( n = 280) hospitalized patients infected with COVID-19 in South Florida hospital. 173 patients received ivermectin 200 mcg/kg orally and usual clinical care, while 107 patients received the usual clinical care only. …

IFN-γ-driven skewing towards Th1 over Th17 differentiation underlies CRS and neutropenia in CAR-T therapy.

Chimeric antigen receptor T cell (CAR-T) therapy has led to significant improvements in patient survival. However, a subset of patients experience high-grade toxicities, including cytokine release syndrome (CRS) and immune cell-associated hematological toxicity (ICAHT). We utilized IL-2Ra knockout mice to model toxicities with elevated levels of IL-6, IFN-γ, and TNF-α and increased M1-like macrophages. Onset of CRS was accompanied by a reduction in peripheral blood neutrophils due to disruption of bone marrow neutrophil homeostasis characterized by an increase in apoptotic neutrophils and a de

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… MDJ reports grants and personal fees from Kite/Gilead, personal fees from Myeloid Therapeutics, Novartis, and BMS, and grants from Incyte, Loxo@Lilly, FACCA, Mark Foundation, and Bankhead-Coley State of Florida outside the submitted work. …

mHealth technologies in research studying cardiovascular health in cancer: A systematic review.

Cancer survivors face an increased risk of cardiovascular disease (CVD) due to treatment-related toxicity, lifestyle factors, and comorbidities. Addressing CV health is crucial for improving quality of life and long-term outcomes. The American Heart Association's Life's Essential 8 framework highlights modifiable determinants of CV health, emphasizing early detection and monitoring. Mobile health (mHealth) technologies, such as wearables and smartphone apps, offer continuous tracking, yet their applications in cancer survivorship remain unclear. This review systematically characterizes the typ

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… . • Commercial fitness tracker (Fitbit Flex wearable wristband; Fitbit, Inc., San Francisco, California) • Research-Grade Accelerometer (Actigraph GT3X; ActiGraph LLC, Pensacola, Florida) • Phone calls and text messages to support communication | • PA measures derived from the Fitbit included daily steps …

Therapeutic drug monitoring for cytotoxic anticancer drugs: Principles and evidence-based practices.

Cytotoxic drugs are highly efficacious and also have low therapeutic index. A great degree of caution needs to be exercised in their usage. To optimize the efficacy these drugs need to be given at maximum tolerated dose which leads to significant amount of toxicity to the patient. The fine balance between efficacy and safety is the key to the success of cytotoxic chemotherapeutics. However, it is possibly more rewarding to obtain that balance for this class drugs as the frequency of drug related toxicities are higher compared to the other therapeutic class and are potentially life threatening

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… Pharmacotherapy ( 1997 ) 17 ( 5 ): 881–6 . 9324177 89 Highlights from: 5-fluorouracil drug management pharmacokinetics and pharmacogenomics workshop; Orlando, florida; January 2007 . …

Osteoporosis in Men: A Review of an Underestimated Bone Condition.

Osteoporosis is called the 'silent disease' because, although it does not give significant symptoms when it is not complicated, can cause fragility fractures, with serious consequences and death. Furthermore, the consequences of osteoporosis have been calculated to weigh heavily on the costs of health systems in all the countries. Osteoporosis is considered a female disease. Actually, the hormonal changes that occur after menopause certainly determine a significant increase in osteoporosis and the risk of fractures in women. However, while there is no doubt that women are more exposed to osteo

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… An epidemiological study by Sattin et al. [ 29 ] carried out in Miami, Florida, revealed that, in subjects aged 85, the risk of falling is 138.5 per 1000 for men and 156.8 per 1000 for women. …

2021 ACMT Annual Scientific Meeting Abstracts-Virtual.

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… Florida had the single year most cases with 45 in 2006. Eighteen states reported coral snake bites with Florida ( n = 416) and Texas ( n = 359) accounting for 91% of all cases. Nine non-endemic states reported coral snake bites. … Texas had the single year most cases with 77 in 2018. 34 states and the District of Columbia reported cottonmouth bites with Florida ( n = 723), Texas ( n = 459), and Louisiana ( n = 338) reporting the most overall cases. …

Всемирный конгресс молекулярного воображения 2022.

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Proceedings 27th Symposium ESVN‐ECVN

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… Green 3 1 Department of Veterinary Clinical Sciences, The Ohio State University, Columbus, OH, USA 2 Mass Spectrometry and Proteomics Facility, The Ohio State University, Columbus, OH, USA 3 Department of Chemistry, University of Florida, Florida, FL, USA The investigation of the proteins expressed …