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Italian Real-Life Experience of Patients with Hereditary Transthyretin Amyloidosis Treated with Patisiran.

Hereditary transthyretin amyloidosis (ATTRv) is a multisystemic, rare, inherited, progressive and adult-onset disease, affecting the sensorimotor nerves, heart, autonomic function and other organs. The actual scenario of pharmaceutical approaches for ATTRv amyloidosis includes five main groups: TTR stabilizers, TTR mRNA silencers, TTR fibril disruptors, inhibitor of TTR fibril seeding and gene therapy. Patisiran is a small, double-stranded interfering RNA encapsulated in a lipid nanoparticle, able to penetrate into hepatocytes, where it selectively targets TTR mRNA, reducing TTR production. We

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

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Anemia in Chronic obstructive pulmonary disease: Prevalence, pathogenesis, and potential impact.

Chronic obstructive pulmonary disease (COPD) is a common preventable and treatable lifestyle-related disease with high global prevalence. COPD is associated with significant morbidity and mortality worldwide. Comorbidities are important events in the natural history of the disease and have a negative effect on the morbidity and mortality of COPD patients. Cardiac diseases, lung cancer, osteoporosis, and depression are common comorbidities reported for COPD. Recently, anemia has been recognized as a frequent comorbidity in COPD patients. The prevalence of anemia in patients with COPD varies fro

A comprehensive review on pain: Model, target, therapy, and prospect (Review).

Pain serves as both a protective physiological response essential for species survival and a global health issue affecting human well‑being and societal development. The present review systematically summarized and analyzed the research progresses on pain through a multidimensional analytical framework encompassing animal models, molecular targets, therapeutic approaches and future prospects. In animal modeling, neuropathic pain paradigms have evolved from traditional mechanical compression to composite models that combine dynamic displacement and chemical stimulation. Cancer‑induced pain mode

Muscle wasting in cancer: opportunities and challenges for exercise in clinical cancer trials.

BACKGROUND: Low muscle in cancer is associated with an increase in treatment-related toxicities and is a predictor of cancer-related and all-cause mortality. The mechanisms of cancer-related muscle loss are multifactorial, including anorexia, hypogonadism, anaemia, inflammation, malnutrition, and aberrations in skeletal muscle protein turnover and metabolism. METHODS: In this narrative review, we summarise relevant literature to (i) review the factors influencing skeletal muscle mass regulation, (ii) provide an overview of how cancer/treatments negatively impact these, (iii) review factors be

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… Further, the administration of different regimens was associated with differential patterns of muscle loss (all chemotherapy treated animals underwent a loss in quadriceps muscle mass, whereas only animals treated with FOLFIRI experienced a loss in gastrocnemius and tibialis mass). …

Brazilian Nutritional Consensus in Hematopoietic Stem Cell Transplantation: Elderly.

The Brazilian Nutritional Consensus in Hematopoietic Stem Cell Transplantation: Elderly was elaborated by nutritionists, nutrologists and hematologists physicians from 15 Brazilians reference centers in hematopoietic stem cell transplantation, in order to emphasize the importancy of nutritional status and the body composition during the treatment, as well as the main characteristics related to patient's nutritional assessment. Establishing the consensus, we intended to improve and standardize the nutritional therapy during the hematopoietic stem cell transplantation. The Consensus was approved

Acupuncture in the treatment of chemotherapy-induced peripheral neuropathy: a meta-analysis and data mining.

BackgroundThe efficacy and acupoint selection of acupuncture in treating chemotherapy-induced peripheral neuropathy (CIPN) remain controversial. This study aims to explore the specific efficacy and acupoint selection of acupuncture in treating CIPN through a meta-analysis and data mining.MethodsSearching for clinical trials on acupuncture treatment for CIPN in 8 databases, evaluating its efficacy and safety through a meta-analysis, and exploring its acupoint selection through data mining.ResultsThe meta-analysis included 21 studies and 2,121 patients, showing that compared with the control gro

Haematological and Oncological Training Therapy With Stationary Strength and Cardio Machines (HOT) in Routine Cancer Care: A 3-Year Real-World Evaluation of Acceptance, Feasibility, Safety, and Effects.

BackgroundExercise improves physical function and health-related quality of life (HRQoL) in patients with cancer; however, nationwide structured programmes remain limited. A hospital in Germany implemented a donation-funded Haematological and Oncological Training Therapy with Stationary Strength and Cardio Machines (HOT), and a 3-year real-world data analysis aimed to evaluate its acceptance, feasibility, safety, and effects.MethodsHOT comprised 24 sessions of combined resistance and aerobic training, complemented by a sensorimotor module for patients with chemotherapy-induced peripheral neuro

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… Paixão , et al., “ Minimal Clinically Important Difference for Quadriceps Muscle Strength in People With COPD Following Pulmonary Rehabilitation ,” COPD: Journal of Chronic Obstructive Pulmonary Disease 18 , no. 1 ( 2021 ): 35 – 44 . 33533285 10.1080/15412555.2021.1874897 48 R. M. …

Skeletal organoids.

The skeletal system, composed of bones, muscles, joints, ligaments, and tendons, serves as the foundation for maintaining human posture, mobility, and overall biomechanical functionality. However, with ageing, chronic overuse, and acute injuries, conditions such as osteoarthritis, intervertebral disc degeneration, muscle atrophy, and ligament or tendon tears have become increasingly prevalent and pose serious clinical challenges. These disorders not only result in pain, functional loss, and a marked reduction in patients' quality of life but also impose substantial social and economic burdens.

Diagnostic and prognostic value of cardiac imaging in amyloidosis.

Amyloidosis is an infiltrative disease caused by extracellular protein deposition that has accumulated a lot of scientific production in recent years. Different types of amyloidosis can affect the heart. Transthyretin amyloidosis and light chain amyloidosis are the two most common types of cardiac amyloidosis. These entities have a poor prognosis, so accurate diagnostic techniques are imperative for determining an early therapeutic approach. Recent advances in cardiac imaging and diagnostic strategies show that these tools are safe and can avoid the use of invasive diagnostic techniques to his

Влияние краткосрочной прогрессивной физической тренировки на активность заболевания у пациентов с подозрительной множественной миеломой и моноклональной гамма-патологией неопределенного значения: односторонний пилотный исследование.

ОБЪЕКТ: Высокий уровень физической активности связан с уменьшением риска развития крови опухоли множественной миеломы (ММ). ММ предшествует асимптоматическим стадиям моноклональной гамма-патологии неопределенного значения (MGUS) и дымящей множественной миеломы (SMM), которые клинически управляются методом наблюдения. Случайный исследователь (N = 1) бывшего высшего спортсмена в возрасте 44 лет показал, что многофакторная программа физических упражнений обратила активность SMM. С учетом результатов этого предыдущего тематического исследования в настоящем экспериментальном исследовании сначала ра

Effects of Physical Cues on Stem Cell-Derived Extracellular Vesicles toward Neuropathy Applications.

The peripheral nervous system undergoes sufficient stress when affected by diabetic conditions, chemotherapeutic drugs, and personal injury. Consequently, peripheral neuropathy arises as the most common complication, leading to debilitating symptoms that significantly alter the quality and way of life. The resulting chronic pain requires a treatment approach that does not simply mask the accompanying symptoms but provides the necessary external environment and neurotrophic factors that will effectively facilitate nerve regeneration. Under normal conditions, the peripheral nervous system self-r

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

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Low-Level Laser Therapy in the Management of Bisphosphonate-Related Osteonecrosis of the Jaw.

Background: Bisphosphonate-Related Osteonecrosis of the Jaw (BRONJ) is a severe complication associated with bisphosphonate therapy, commonly used in the treatment of osteoporosis and metastatic bone diseases. Low-Level Laser Therapy (LLLT) has been proposed as a potential treatment modality for BRONJ, with its anti-inflammatory, analgesic, and regenerative effects being of particular interest. This systematic review aims to critically assess the current evidence regarding the efficacy of LLLT in the management of BRONJ. Methods: This review was conducted following the PRISMA (Preferred Report

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… Assessment of Quadriceps Muscle in Advanced Knee Osteoarthritis and Correlation with Lower Limb Alignment. Medicina. 2024;60:1983. doi: 10.3390/medicina60121983. 11. Baran C., Kayan A., Baran C.S., Karacuha A.F., Eryilmaz S. …

Cementoplasty to cryoablation: review and current status.

Recent advances in percutaneous image-guided techniques have empowered interventional radiologists with diverse treatment options for the management of musculoskeletal lesions. Of note, there is growing utility for cementoplasty procedures, with indications ranging from stabilization of bone metastases to treatment of painful vertebral compression fractures. Likewise, cryoablation has emerged as a viable adjunct in the treatment of both primary and secondary bone and soft tissue neoplasms. These treatment options have been progressively incorporated into the multidisciplinary approach to holis

An Individualized Exercise Intervention for People with Multiple Myeloma-Study Protocol of a Randomized Waitlist-Controlled Trial.

People with multiple myeloma (MM) are second only to people with lung cancer for the poorest reported health-related quality of life (HRQoL) of all cancer types. Whether exercise can improve HRQoL in MM, where bone pain and lesions are common, requires investigation. This trial aims to evaluate the efficacy of an exercise intervention compared with control on HRQoL in people with MM. Following baseline testing, people with MM (n = 60) will be randomized to an exercise (EX) or waitlist control (WT) group. EX will complete 12-weeks of supervised (24 sessions) and unsupervised (12 sessions) indiv

Advances in delivery methods of Arthrospira platensis (spirulina) for enhanced therapeutic outcomes.

Arthrospira platensis (A. platensis) aqueous extract has massive amounts of natural products that can be used as future drugs, such as C-phycocyanin, allophycocyanin, etc. This extract was chosen because of its high adaptability, which reflects its resolute genetic composition. The proactive roles of cyanobacteria, particularly in the medical field, have been discussed in this review, including the history, previous food and drug administration (FDA) reports, health benefits and the various dose-dependent therapeutic functions that A. platensis possesses, including its role in fighting against

A 16-week progressive exercise training intervention in treatment-naïve chronic lymphocytic leukaemia: a randomised-controlled pilot study.

BackgroundChronic lymphocytic leukaemia (CLL) typically presents with asymptomatic, early-stage disease that is monitored until disease progression ('treatment-naïve' CLL). The objective of this pilot study was to assess the feasibility and preliminary safety of an exercise program in treatment-naïve CLL. We also sought to preliminarily assess the impact of the exercise program on disease activity, as it has been proposed that exercise training may reduce disease outgrowth in treatment-naïve CLL.MethodsA total of 40 treatment-naïve CLL patients were recruited into this randomised-controlled pi

CRLF1 and CLCF1 in Development, Health and Disease.

Cytokines and their receptors have a vital function in regulating various processes such as immune function, inflammation, haematopoiesis, cell growth and differentiation. The interaction between a cytokine and its specific receptor triggers intracellular signalling cascades that lead to altered gene expression in the target cell and consequent changes in its proliferation, differentiation, or activation. In this review, we highlight the role of the soluble type I cytokine receptor CRLF1 (cytokine receptor-like factor-1) and the Interleukin (IL)-6 cytokine CLCF1 (cardiotrophin-like cytokine fa

Molecular and cellular mechanisms of skeletal muscle atrophy: an update.

Skeletal muscle atrophy is defined as a decrease in muscle mass and it occurs when protein degradation exceeds protein synthesis. Potential triggers of muscle wasting are long-term immobilization, malnutrition, severe burns, aging as well as various serious and often chronic diseases, such as chronic heart failure, obstructive lung disease, renal failure, AIDS, sepsis, immune disorders, cancer, and dystrophies. Interestingly, a cooperation between several pathophysiological factors, including inappropriately adapted anabolic (e.g., growth hormone, insulin-like growth factor 1) and catabolic pr

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… For instance, in symptomatic CHF patients, quadriceps muscle strength is reduced compared to controls, even after correction for reduced circumferential area [ 8 ]. Equally important, however, are the prognostic implications of cachexia and muscle loss. …