A5091435937- Muscle Physiology and Disorders
- Neurogenetic and Muscular Disorders Research
- Prosthetics and Rehabilitation Robotics
- Health Systems, Economic Evaluations, Quality of Life
- Congenital Anomalies and Fetal Surgery
- Genetic Neurodegenerative Diseases
- Multiple Sclerosis Research Studies
- Muscle activation and electromyography studies
- Cerebral Palsy and Movement Disorders
- Bone health and osteoporosis research
- Family and Disability Support Research
- Fibromyalgia and Chronic Fatigue Syndrome Research
- Cardiomyopathy and Myosin Studies
- Parkinson's Disease Mechanisms and Treatments
- Bone health and treatments
- Biomedical Ethics and Regulation
- Connective tissue disorders research
- Lymphoma Diagnosis and Treatment
- Childhood Cancer Survivors' Quality of Life
- Hip disorders and treatments
- Systemic Sclerosis and Related Diseases
- Delphi Technique in Research
- Genetics, Aging, and Longevity in Model Organisms
- Advanced Causal Inference Techniques
- Lung Cancer Treatments and Mutations
IQ Samhällsbyggnad
2022-2025
Karolinska Institutet
2014-2023
IQVIA (United Kingdom)
2022-2023
Children's Hospital of Eastern Ontario
2023
Imperial Consultants
2019
Innovation Management Consulting Group (Sweden)
2016
Optum (United States)
2014
GlaxoSmithKline (United Kingdom)
2014
GlaxoSmithKline (Sweden)
2014
Newcastle University
2014
The objective of this study was to estimate the total cost illness and economic burden Duchenne muscular dystrophy (DMD).Patients with DMD from Germany, Italy, United Kingdom, States were identified through Translational Research in Europe-Assessment & Treatment Neuromuscular Diseases registries invited complete a questionnaire online together caregiver. Data on health care use, quality life, work status, informal care, household expenses collected costs perspective society caregiver...
Duchenne muscular dystrophy (DMD) is a rare pediatric neuromuscular disease associated with progressive muscle degeneration and extensive care needs. Our objective was to estimate the caregiver burden DMD. We made cross-sectional assessments of health-related quality life (HRQL) using EuroQol EQ-5D, Visual Analogue Scale (VAS), SF-12 Health Survey, Zarit Caregiver Burden Interview (ZBI) administered online. Results were stratified by stage (early/late ambulatory/non-ambulatory) caregivers'...
To estimate health-related quality of life (HRQOL) in patients with Duchenne muscular dystrophy (DMD).HRQOL was assessed using the Health Utilities Index Questionnaire (HUI) and Pediatric Quality Life Inventory (PedsQL) neuromuscular module version 3.0 online. Results were stratified by disease stage (early/late ambulatory/non-ambulatory) caregivers' perceptions patients' health mental status.A total 770 patient-caregiver pairs (173 German, 122 Italian, 191 UK, 284 USA) participated. Most...
Background: International care guidelines for Duchenne muscular dystrophy (DMD) were published in 2010, but compliance clinical practice is unknown. Objective: The objective of our study was to compare real-world DMD Germany, Italy, the UK
Achondroplasia, caused by a pathogenic variant in the fibroblast growth factor receptor 3 gene, is most common skeletal dysplasia. The Lifetime Impact of Achondroplasia Study Europe (LIAISE; NCT03449368) aimed to quantify burden achondroplasia among individuals across broad range ages, including adults.Demographic, clinical and healthcare resource use data were collected from medical records patients enrolled 13 sites six European countries this retrospective, observational study....
Costs of informal care account for a significant component total societal costs many chronic and disabling illnesses. Yet, associated with the provision is seldom included in economic evaluations new health technologies, increasing risk suboptimal decisions on allocation resources from perspective society. Our objective was to propose standardized questionnaire measurement, valuation, estimation caregiver indirect (productivity) as separate mutually exclusive subsets cost-of-illness studies...
The objective of this retrospective, observational study was to estimate the long-term impact early treatment multiple sclerosis (MS) on risk disability pension. Our cohort comprised patients with MS in Sweden, identified a nationwide disease-specific register (the Swedish Multiple Sclerosis Registry), who started disease-modifying drug (DMD) between January 1, 2002, and December 31, 2012. We analyzed association time from onset initiation full-time pension using survival analysis. sample...
Background: Multiple sclerosis (MS) is associated with considerable morbidity and serious disability, but little known of the long-term impact disease on work ability. Objectives: To assess sick leave (short-term absence) disability pension (long-term before after diagnosis MS. Methods: Patients MS in Sweden were identified a nationwide disease-specific register matched general population controls. Sick measured index (i.e. date). Results: The final sample comprised 6092 patients 60,345...
The objective of this cross-sectional, observational study was to investigate the disease burden myotonic dystrophy type 1 (DM1), a disabling muscle disorder.
Abstract Background Management and treatment of spinal muscular atrophy (SMA) has changed in recent years due to the introduction novel transformative potentially curative therapies resulting emergence new disease phenotypes. Yet, little is known about uptake impact these real-world clinical practice. The objective this study was describe current motor function, need assistive devices, therapeutic supportive interventions provided by healthcare system, as well socioeconomic situation...