Musculoskeletal Diseases

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping

The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

This randomized clinical trial (RCT) aims to compare clinical and radiographic outcomes of different partial wrist fusion techniques in participants with post-traumatic wrist arthritis. Participants with stage II or III scapholunate advanced collapse (SLAC) or scaphoid nonunion advanced collapse (SNAC) who meet the eligibility criteria will be randomly assigned to one of two parallel groups: Group A (partial wrist arthrodesis without triquetral excision i.e. four-corner arthrodesis), or Group B (partial wrist arthrodesis with triquetral excision i.e. three-corner or capitolunate arthrodesis with triquetral excision). The results of this study will provide evidence to guide surgeons in determining the ideal wrist fusion technique in the management of patients with post-traumatic wrist arthritis requiring surgery.

The 100-Year Human Aging Study is a prospective, pragmatic, observational trial enrolling participants across fixed and mobile clinical sites to undergo comprehensive multi-system health screening and longitudinal follow-up until death. Participants are followed to determine whether measurements taken at enrollment and repeated across the lifespan - individually and in combination - predict all-cause mortality, cause-specific mortality, incident serious disease, and functional disability. The study is designed to generate the surrogate endpoint validation data that longevity medicine currently lacks.

The primary objective of this study is to translate the Nordic Musculoskeletal (E-NMQ) Questionnaire Modified Version into Urdu following international guidelines. Presently, there exists no validated tool to comprehensively measure symptoms such as pain, stiffness and weakness in Urdu-speaking patients in Pakistan. Specifically, the widely used E-NMQ, having these three vital categories, has yet to be translated and validated for Urdu-speaking individuals with musculoskeletal disease (MSD).

This study, in a quasi-experimental matched two-group pre-post design, investigates the effect of serial water-filtered whole-body hyperthermia on circadian core body temperature, the somatosensory system (nociception) and the movement evoked pain in healthy and patients with chronic primary pain (e.g., fibromyalgia). The intervention lasts 3 weeks with two treatment sessions per week.

Adults are often encouraged to exercise to maintain or improve bone health. However, there is evidence that exercise does not always lead to increases in bone mass, and exercise could lead to bone loss under certain conditions. Endurance exercise can increase bone resorption following an exercise bout, which may explain why bone does not always favorably adapt to exercise, but it is unclear if this also happens with resistance exercise. Further, it is not known how exercise training influences blood markers of bone resorption for either endurance or resistance exercise. The purpose of this study is to determine 1) if resistance exercise causes a similar increase in bone resorption as endurance exercise; and 2) if exercise training influences the increase in bone resorption following exercise for both endurance and resistance exercise.

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping

The purpose of this study is to develop a multidimensional screening tool for Work-Related Musculoskeletal Disorders (WMSDs) and to evaluate its psychometric properties. The study involves a cross-sectional survey of approximately 250 workers in the bio-pharmaceutical industry to assess the tool's structural validity, internal consistency, construct validity, and measurement invariance following COSMIN guidelines.

This study aimed to assess the prevalence of MSDs and the level of ergonomics knowledge among dentists who are specializing in different branches or have completed specialist training.

The aim of this study is to compare ultrasound-guided perineural injection of the median nerve with classic minimal incision surgical technique for median nerve decompression in patients diagnosed with mild, moderate, and severe carpal tunnel syndrome.

The aim of our study is to determine the relationship between disease severity and sleep quality in CTS patients and to compare the findings with healthy controls.

This study aims to investigate the relationship between physical activity levels and posture, sleep quality, depression, and musculoskeletal system problems among academic staff working in the health sciences field at Istanbul Aydın University. Modern technological developments and sedentary work patterns have significantly reduced daily physical activity levels. Academic personnel often spend long hours sitting, preparing lectures, conducting research, and using computers, which may negatively affect posture and increase the risk of musculoskeletal pain. Reduced physical activity has also been associated with poor sleep quality and increased depressive symptoms. This cross-sectional study will include academic staff aged 23-65 years working in the Faculty of Health Sciences. Participation will be voluntary. Data will be collected using a sociodemographic information form and validated assessment tools, including the International Physical Activity Questionnaire-Short Form (IPAQ), New York Posture Rating Chart, Pittsburgh Sleep Quality Index (PSQI), Beck Depression Inventory (BDI), and the Nordic Musculoskeletal Questionnaire. The primary objective is to evaluate whether physical activity levels are associated with posture, sleep quality, depression levels, and musculoskeletal complaints. The findings are expected to contribute to the development of preventive strategies aimed at improving occupational health, well-being, and quality of life among academic personnel.

Currently the diagnosis of OA is based on radiographs and clinical findings, which is limited to detecting late-stage disease. There is a pressing, unmet clinical need for robust assessment of early changes in cartilage health. Towards this goal, extensive efforts are ongoing to develop quantitative MRI for cartilage matrix analysis. MR T1ρ and T2 relaxation times have shown to be promising imaging biomarkers for early cartilage degeneration and prediction of disease progression. However, many challenges remain to clinically applying these techniques, including lack of standardized acquisition and quantification methods, and long acquisition times. The study aims to develop novel, fast and reproducible MR T1ρ and T2 relaxation time imaging methods on MR systems from multiple vendors and establish a platform for standardization and cross validation of these measures as a tool for clinical trials using such techniques. Following method validation, patients at risk for osteoarthritis will be tested.

Breast cancer patients who receive endocrine therapy particularly aromatase inhibitors often experience aromatase inhibitors associated symptoms (AIMSS) such as joint and muscle pain along with stiffness and fatigue that can disrupt with daily activities and lead some patients to stop treatment early. A structured intervention program named AIMSS-CARE (Aromatase Inhibitor-associated Musculoskeletal Symptoms-Comprehensive Adapted Rehabilitation Evaluation) developed in China that combines exercise, education, symptom monitoring, and follow-up has been shown to reduce these symptoms and improve treatment adherence. This study will adapt this program for use in Ethiopia while testing its effectiveness to enhance pain management, treatment adherence and quality of life among Ethiopian breast cancer patients receiving endocrine therapy. The study will be conducted at Tikur Anbessa Specialized Hospital in Addis Ababa, Ethiopia. Eighty-eight patients will be randomly assigned to either the adapted intervention program or usual care. The research will also examine the feasibility and acceptability of the intervention to patients and healthcare providers, and what factors influence its successful implementation. Results will help determine whether this program can be used more widely in Ethiopia and other African cancer centers.

Developing and testing a musculoskeletal national audit in community/primary care

The proposed study evaluates the effect of ankle foot orthosis (AFO) heel height and stiffness on the forces and motion of the lower limb during over-ground walking in individuals who use an AFO for daily walking. Previous studies suggest that heel height and stiffness effect limb loading, but these data and the analysis techniques applied are limited. In this study, heel cushions with different height and stiffness's (4 conditions) will be placed in participants shoes and they will walk at controlled and self-selected speeds. Participants will also walk with their AFO as configured prior to enrollment, and with no AFO if possible. The proposed study will provide evidence that can be used by clinicians and researchers to align braces that most effectively improve function during every-day walking.

The EULAR Impact of RMDs Survey collects first-hand information about the impact of rheumatic and musculoskeletal diseases (RMDs) among patients across and beyond Europe. Through periodic questionnaires, patients will provide information about their healthcare situation, and how the disease affects their social and occupational lives. This data will be an important resource for researchers, healthcare professionals, and patients alike, providing valuable insights into the burden of disease and helping improve the overall care for people living with these conditions.

The goal of this study is to investigate the impact of a home-based shoulder strength training on the overall shoulder health in manual wheelchair users, and if the timepoint of such a training makes a difference. The study is focusing on persons with a spinal cord injury in the thoracic or lumbar region of the spine, that have only recently been injured and will soon be discharged from primary rehabilitation. The shoulder training will take place either 3 or 12 months after discharge from primary rehabilitation and will be carried out twice a week for 12 weeks. There are six measurements occurring every three months, which leads to an overall duration of 15 months. The measurements consist of: * Questionnaires about independence in daily life, participation, quality of life and physical activity * Assessment of shoulder strength, range of motion and function * Measurement of the daily wheelchair use during one week via sensors that are fixed to the wheelchair and wrist * one further questionnaire at the end of the measurement week about the occurrence of shoulder pain Additionally, on four of the six measurement timepoints, a magnet resonance image (MRI) of the shoulder will be taken to assess the shoulder status (pathology, muscle volume and quality). Though all these measurements the researchers can additionally assess the load of daily life on the shoulders, and how well this matches the preparation during the primary rehabilitation.

This is a prospective, single center, double arm, randomized, unmasked, First in Human study that aims to evaluate the safety and effectiveness of SakuraBead™ resorbable embolization microspheres in adult patients suffering from pain secondary to plantar fasciitis.

Lipedema is a common disease of subcutaneous adipose tissue. The most common complaint of patients with swelling in the affected extremity is pain. In addition, patients with lipedema may experience conditions that can greatly affect the health and quality of life of the individual, such as loss of muscle strength and exercise capacity, and deterioration in activity levels of daily living. It is still unknown whether the decrease in muscle strength in patients with lipedema is part of this condition or whether decreased activity levels lead to decreased muscle strength. Sarcopenia is an important health problem characterized by age-related loss of muscle mass and muscle function. The relationship between muscle weakness and sarcopenia in patients with lipedema has not been investigated before. Early recognition of possible sarcopenia and functional limitations in these patients may be important to increase the ability of patients to participate in physical activity as part of their conservative management. There is no study in the literature investigating sarcopenia in patients with a diagnosis of lipedema. The aim of this study is to evaluate patients with a diagnosis of lipedema in terms of sarcopenia. In addition, the relationship between sarcopenia and age, body mass index, exercise frequency, lipedema type, and stage will be investigated in patients diagnosed with lipedema.

Tuberculosis (TB) is one of the top ten causes of death worldwide with approximately 10 million cases globally and 1.2 million deaths. Sub-Saharan Africa carries the highest burden of TB. South Africa has one of the highest HIV and TB rates worldwide with an HIV prevalence rate in adults of 19% and a TB case notification rate of 615/100,000 in 2019. Over many years, focus has been paid to pulmonary TB and extrapulmonary TB (EPTB) has received only little attention even though it accounts for almost a quatre of all TB cases. The diagnosis of EPTB remains challenging simply because sample collection requires invasive procedures in the absence of a blood-based diagnostic test. Spinal TB (spondylitis or spondylodiscitis caused by Mycobacterium tuberculosis) - often known as Pott's disease - accounts for up to 10% of EPTB and affects young children, people with HIV-coinfection and elderly, and often leads to lifelong debilitating disease due to devastating deformation of the spine and compression of neural structures. Little is known with regards to the extent of disease and isolated TB spine as well as a disseminated form of TB spine have been described. The latter presents with a spinal manifestation plus disseminations to other organs such as the lungs, pleura, lymph nodes, the GIT or urinary tract or even the brain. In the Spinal TB X cohort, the investigators aim to describe the clinical phenotype of spinal TB using whole body PET/CT and identify a specific gene expression profile for the different stages of dissemination and compare findings to previously described signatures for latent and active pulmonary TB. A blood-based test for spinal TB would lead to earlier diagnosis and treatment in all settings globally and improve treatment outcome of this devastating disease.

This study examines the health outcomes of patients with long-term illnesses such as cancer, heart, lung, or musculoskeletal diseases who participate in a rehabilitation programme. The programme may involve staying in a facility (inpatient), visiting a facility (outpatient), home-based care supported by digital health tools (telerehabilitation), or a combination of facility- and home-based options. The key health outcomes being measured include physical fitness and strength, body composition (like body fat and muscle mass), specific risk factors related to the diseases, and patients' self-reported health and experience outcomes, such as feelings of anxiety, depression, fatigue, overall quality of life, and satisfaction with the rehabilitation programme. These measurements provide important information about the patients' lung, heart, and muscle health, as well as their mental well-being. They also help to predict the likelihood of further health complications, the chance of needing to be hospitalized again, and overall quality of life. This information is useful for improving the quality of care, tailoring future provision of rehabilitation services and long-term care for patients with chronic conditions. Additionally, the study will provide insights into how new technologies like telerehabilitation are implemented, including how well they are adopted, followed, and accepted by patients.

People with Parkinson's have infrequent clinical consultation (once every 12-18 months) and limited rehabilitation. Assessment play an important role in these consultations to help clinicians understand patients' health status and disease progression necessary to adjust treatment plans. The current way of measuring is the UPDRS which needs a clinician to do this and takes 30 minutes. There is a strong need for more frequent and accurate Parkinson's assessments in the clinic and at home to detect changes early and then give appropriate support and drug and physiotherapy quickly. There is a need to develop good home digital physiotherapy tools to increase the amount of therapy. Here the investigators are testing new digital technologies to do these assessments in the home and clinic and a new digital physiotherapy device in the home. The investigators aim to conduct a clinical study with 50 people with Parkinson's (50 from UK) with the UPDRS, (a rating scale that is commonly used in clinical settings to evaluate the progression of Parkinson's disease) and 30 healthy adults. The investigators will develop and investigate if two new digital devices, one the MachineMD that measures eye movement and one the gaitQ that measures gait can be used instead of the MDS-UPDRS (motor) using digital gait and ophthalmic features in the clinic setting. The investigators will investigate the effect of a physiotherapy gait intervention gaitQ Tempo in the home context for two weeks and of doing the gait measure at home. The investigators will determine the potential of the gaitQ intervention to improve key gait metrics in order to collect clinical evidence and of using the gaitQ as a cuing system over a 2-week period on gait and other movement measures in the home and community

The aim of this observational study is to validate the content of a tailored ICF core set for the management of musculoskeletal conditions in primary care physiotherapy services. The main question it aims to answer is: \- Are the ICF entities included in the ICF Core Set under evaluation useful from a clinical point of view? Participants will evaluate the relevance of each ICF entity included in the ICF core set according to their musculoskeletal condition.