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Chemical Proteomics and Phenotypic Profiling Identifies the Aryl Hydrocarbon Receptor as a Molecular Target of the Utrophin Modulator Ezutromid.

Duchenne muscular dystrophy (DMD) is a fatal muscle-wasting disease arising from mutations in the dystrophin gene. Upregulation of utrophin to compensate for the missing dystrophin offers a potential therapy independent of patient genotype. The first-in-class utrophin modulator ezutromid/SMT C1100 was developed from a phenotypic screen through to a Phase 2 clinical trial. Promising efficacy and evidence of target engagement was observed in DMD patients after 24 weeks of treatment, however trial endpoints were not met after 48 weeks. The objective of this study was to understand the mechanism o

Health and aging in elderly farmers: the AMI cohort.

BackgroundThe health of the agricultural population has been previously explored, particularly in relation to the farming exposures and among professionally active individuals. However, few studies specifically focused on health and aging among elders retired from agriculture. Yet, this population faces the long-term effects of occupational exposures and multiple difficulties related to living and aging in rural area (limited access to shops, services, and practitioners). However, these difficulties may be counter-balanced by advantages related to healthier lifestyle, richer social support and

Patient-reported data informing early benefit assessment of rare diseases in Germany: A systematic review.

BackgroundSince the implementation of the Regulation on Patient Integration (2003), the Act on the Reorganization of the Pharmaceutical Market (2011), and the Patient Rights Law (2013), the inclusion of patient perspectives has been further anchored in the German early benefit assessment process. During the assessment of rare disease interventions, patient perspectives are particularly important, as clinical studies are often designed acknowledging small samples and patients suffering from severe symptoms and the chronic course of the disease. Therefore, our research question is whether patien

Изучение перспектив потребителей услуг в области здравоохранения на пути к интеграции питания в рутинную помощь с множественным склерозом: качественное исследование.

КОЛИЧЕСТВО: Люди, страдающие множественным склерозом, рассчитывают получить информацию о питании от медицинских работников (HCP), с тем чтобы самостоятельно справиться со своим заболеванием. Цель этого исследования заключалась в изучении опыта ПМСО в области обеспечения ухода, связанного с питанием, и в обеспечении понимания их потребностей и предпочтений в отношении того, каким образом его можно было бы обеспечить в рамках обычного ухода. МЕТОДЫ: Это качественное исследование, опирающееся на интерпретационный феноменологический подход, было проведено с использованием онлайновых целевых групп

Chronic Pain and Bone-Related Pathologies: A Narrative Review.

PurposePain related to bone may occur as a result of trauma, bone fracture, genetic disease, arthritis, benign or malignant primary bone tumors and bone cancer metastases. We discuss the pathophysiology of chronic bone-related pain, treatment options and therapeutic perspectives.MethodsUsing predefined terms, we searched PubMed, MEDLINE, and Google Scholar for meta-analyses, evidence-based reviews, and clinical practice guidelines. This narrative article reviews pathologies linked to chronic bone pain and discusses the preventive and therapeutic strategies for better bone pain management.Resul

Are the Physical Environments of Treatment Centres Meeting Recommendations for Patient-Centred Care? Perceptions of Haematological Cancer Patients.

The physical environment of a treatment centre may impact the well-being of patients and their perceptions of care. Outpatients with haematological cancer may be in contact with the treatment centre over long periods and could be particularly affected. This study aimed to identify haematological cancer patients' perceptions of supportive design elements in the hospital they attended and associations with self-reported mood or well-being. Outpatients from three large metropolitan hospitals in Australia were mailed a self-report questionnaire and responded to statements about the treatment centr

Whole-body Computed Tomography Versus Dual Energy X‑ray Absorptiometry for Assessing Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva.

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder that leads to heterotopic ossification (HO), resulting in progressive restriction of physical function. In this study, low-dose, whole-body computed tomography (WBCT) and dual energy X-ray absorptiometry (DXA) were evaluated to determine the preferred method for assessing total body burden of HO in patients with FOP. This was a non-interventional, two-part natural history study in patients with FOP (NCT02322255; date of registration: December 2014). In Part A (described here), WBCT and DXA scans were individually ass

The role of RANK-ligand inhibition in cancer: the story of denosumab.

The diagnosis of bone metastases is an event with certain consequences for the patient. They often mean pain and can also mean pathological fractures, hypercalcemia, and spinal cord compression, all synonymous with a diminished quality of life and often also hospitalization. Since the advent of the intravenous bisphosphonates, things began to look a bit brighter for patients with bone metastases-bone destruction was kept at bay a little longer. The next generation of bone metastasis treatments is well on its way in clinical development, and among them, the most advanced drug is denosumab. Deno

Health- related quality of life after surgery for spinal metastases.

BACKGROUND: Surgery for spinal metastases is almost always palliative and part of a multidisciplinary approach that has determined a significant increase of life expectancy in the last decade; thus, an improvement in health-related quality of life (HRQOL) is the main goal of the treatment of spinal metastases. We report here the results of a prospective study conducted with the aim of evaluating the impact of spinal surgery on HRQOL, measured by Patient-Reported Outcomes Measures (PROMs). We analyzed clinical outcomes (ambulatory status, performance status, pain, neurological status) and HRQOL

Nontraumatic Spinal Cord Injury: Epidemiology, Etiology and Management.

The spinal cord is a conduit within the central nervous system (CNS) that provides ongoing communication between the brain and the rest of the body, conveying complex sensory and motor information necessary for safety, movement, reflexes, and optimization of autonomic function. After a traumatic spinal cord injury (SCI), supraspinal influences on the peripheral nervous system and autonomic nervous system (ANS) are disrupted, leading to spastic paralysis, sympathetic blunting, and parasympathetic dominance, resulting in cardiac dysrhythmias, systemic hypotension, bronchoconstriction, copious re

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… Utilizing the needs assessment checklist, Kennedy and Chessell were able to provide evidence in favor of the NTSCI group for greater increases in activities of daily living, bowel management, community preparedness, and effective use of wheelchairs and equipment [ 81 ]. …

The unseen symptom: A longitudinal qualitative interview study exploring mobility loss in people with advanced cancer.

BACKGROUND: Mobility loss is a common symptom in people with advanced cancer and is associated with reduced function and quality of life. While its physical consequences are well documented, less is known about how individuals and their families experience and adapt to these changes over time. AIM: To explore how individuals with advanced cancer and their caregivers experience and respond to mobility loss. DESIGN: A qualitative longitudinal study using Interpretative Phenomenological Analysis. Participants completed two semi-structured interviews, with follow-ups prompted by changes in mobil

Предсказатели высоко- и низкоинтенсивного лечения склероза первой линии.

КОЛИЧЕСТВО: Лекарства для лечения различных склерозов (MS) могут быть либо с высокой, либо с низкой интенсивностью. Цель этого исследования заключается в изучении прогнозов, связанных с высокоинтенсивными стратегиями обработки MS с использованием данных о коммерческих претензиях. Мы использовали коммерческие и дополнительные базы данных " Merative MarketScan " для обследования пациентов с помощью: амбулаторного диагноза MS ( " Merative MarketScan " )ICD-9/-10:: 340xx, G35.xx); заполнение ДМТ; отсутствие ДМТ 1 год, предшествующий диагностике MS с помощью DMT; и 6 Непрерывная

Взаимосвязь между активными шагами и падающими психологическими факторами при множественном склерозе.

Цель: Лица с множественным склерозом (МС) сталкиваются с изменениями в балансе, такими, как плохие и ответные шаги, а также меняющиеся психологические факторы, связанные с падением, такие, как растущая обеспокоенность по поводу падения и опасения последствий падения. Такие опасения и страх могут быть связаны с мобильностью и влиять на нее. Однако эти отношения плохо понимаются людьми с MS. Цель этого исследования заключалась в изучении связи между реактивными действиями и осенними психологическими факторами, а также в оценке того, как эти отношения различаются между лицами, которые имели и не

Клиническое и экономическое воздействие работников, обеспечивающих уход за пациентами, страдающими множественным склерозом, в зависимости от состояния болезни.

Справочная информация: Исследования по вопросу о партнерах по уходу за пациентами с множественным склерозом (МС) являются ограниченными. Цели: Клиническое и экономическое воздействие на партнеров по уходу за работниками оценивалось с точки зрения степени заболеваемости MS. Методы: работники с супругами/домашними партнерами с НР из базы данных о партнерах по работе (Jan. 1, 2010- Декабрь. 31, 2019) имеют право на получение пособия, если: супруг/партнер имел по крайней мере 3 Связанная с НРICD-9-CM/ICD-10-CM::340xx/G35) заявления пациентов/акушеров/болезней в рамках 1 год (последняя претензия =

Trends in surgical management of spinal metastases in a Singaporean tertiary referral center: a 17-year retrospective review.

IntroductionSurgical treatment is increasingly the treatment of choice in cancer patients with epidural spinal cord compression and spinal instability. There has also been an evolution in surgical treatment with the advent of minimally invasive surgical (MIS) techniques and separation surgery. This paper aims to investigate the changes in epidemiology, surgical technique, outcomes and complications in the last 17 years in a tertiary referral center in Singapore.MethodsThis is a retrospective study of 383 patients with surgically treated spinal metastases treated between January 2005 to January

'Unpacking' pathways to lymphoma and myeloma diagnosis: Do experiences align with the Model of Pathways to Treatment? Findings from a UK qualitative study with patients and relatives.

ObjectivesTo explore alignment of experiences before lymphoma and myeloma diagnosis with the appraisal, help seeking and diagnostic intervals in the Model of Pathways to Treatment (MPT).DesignA qualitative study using in-depth semistructured interviews with patients and relatives. Interviews were transcribed verbatim, anonymised and analysed using qualitative description.SettingA UK population-based haematological malignancy patient cohort.ParticipantsFifty-five patients (35 lymphoma, 20 myeloma: diagnosed 2014-2016) and 28 relatives participated, within around a year of the patient's diagnosi

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… Others used sticks, wheelchairs or riser chairs to remain mobile. …

Меры по согласованию и износостойкости в рамках системы многоразового склероза-системного обзора доказательств и применимости используемых систем скоринга в клинических и реальных данных.

В случае множественного склероза (MS) все большее внимание уделяется соприкосновениям и слабостям, влияющим на курс болезни, лечение и качество жизни. Цель этого обзора литературы заключается в оценке фактических данных, сильных и слабых сторон инструментов оценки соответствия и слабости у людей с НР (МСУ). МЕТОДЫ: Мы провели всеобъемлющий систематический обзор литературы, включая исследования, опубликованные между 2014 и 2024 с первоначальными данными или систематическими обзорами показателей комординации или слабости в ПТУС с использованием PRISMA 2020 методология. Были проанализированы меры

2,4 Dinitrophenol as Medicine.

In the sanctity of pure drug discovery, objective reasoning can become clouded when pursuing ideas that appear unorthodox, but are spot on physiologically. To put this into historical perspective, it was an unorthodox idea in the 1950's to suggest that warfarin, a rat poison, could be repositioned into a breakthrough drug in humans to protect against strokes as a blood thinner. Yet it was approved in 1954 as Coumadin® and has been prescribed to billions of patients as a standard of care. Similarly, no one can forget the horrific effects of thalidomide, prescribed or available without a prescri

Понимание депрессии у людей, живущих с множественным склерозом: обзор последних событий в литературе.

Множественный склероз (MS) — хроническое нейродегенеративное и аутоиммунное заболевание, которое затрагивает примерно 1 миллион взрослых людей в США. Психологические расстройства являются типичными коморбидами у людей с MS (PwMS), причем наиболее распространенным явлением является депрессия. Клиническая депрессия в ПТМС может оказать существенное воздействие на качество жизни и фактор, в значительной степени влияющий на состояние лечения. Депрессия может вспыхнуть на ранних стадиях множества, что становится все более распространенным по мере развития болезни и усиления степени клинической инва

Safety and efficacy of antigen-specific therapeutic approaches for multiple sclerosis: Systematic review.

INTRODUCTION: The objective of this systematic review is to evaluate the efficacy and safety of antigen-specific tolerance-inducing therapeutic approaches (products based on peptides, DNA and T cells) versus placebo or other comparators, where possible, in adult multiple sclerosis (MS) patients. METHODS: PubMed, CINAHL, Web of Science, Cochrane and International Clinical Trials Registry Platform, ClinicalTrials.gov were searched for published and unpublished studies. Screening, critical appraisal, and data extraction for included studies were carried out by two independent reviewers. For effi

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… To support mobility, patients use mobility assistive technology (MAT), such as wheelchairs, scooters, walkers, canes etc. …