A5005547614- Muscle Physiology and Disorders
- Tissue Engineering and Regenerative Medicine
- Health Policy Implementation Science
- Mental Health and Patient Involvement
- Prosthetics and Rehabilitation Robotics
- Participatory Visual Research Methods
- Neurogenetic and Muscular Disorders Research
- Global Health Care Issues
- Genetics, Aging, and Longevity in Model Organisms
- Health Systems, Economic Evaluations, Quality of Life
- Insurance, Mortality, Demography, Risk Management
- Genetic Associations and Epidemiology
- Parathyroid Disorders and Treatments
- Patient-Provider Communication in Healthcare
- Exercise and Physiological Responses
- Cerebral Palsy and Movement Disorders
- demographic modeling and climate adaptation
- Muscle activation and electromyography studies
- Medical Imaging and Pathology Studies
- Genomics and Rare Diseases
- Healthcare Policy and Management
- Genetic Syndromes and Imprinting
- Telomeres, Telomerase, and Senescence
University of Leicester
2021-2024
Newcastle University
2021-2024
University of Warwick
2023
Critical Path Institute
2023
Karolinska Institutet
2021-2023
GlaxoSmithKline (India)
2023
Primary Source
2023
Leicester College
2023
Cambridge University Hospitals NHS Foundation Trust
2022
Department of Medical Sciences
2021
Clinical trials in Duchenne muscular dystrophy (DMD) require 3-6 months of stable glucocorticoids, and the primary outcome is explored at 48-52 weeks. The factors that influence clinical assessment (COA) trajectories soon after glucocorticoid initiation are relevant for design analysis novel drugs. We describe early COA trajectories, associated factors, time from to peak.
Abstract Primary hyperparathyroidism (PHPT) is characterized by hypercalcemia driven excess parathyroid hormone (PTH) secretion. PHPT a common endocrine condition with prevalence of 1 to 7 cases per 1000 adults. typically presents in the fifth or sixth decade and shows significant female preponderance. Solitary hyperfunctioning adenomas account for 85% 90% cases. The remaining 10% 15% include multiglandular disease (multiple hyperplasia) and, rarely, carcinoma (1%). Ectopic may arise due...
Abstract Background Duchenne muscular dystrophy (DMD) is a rare, muscle‐degenerative disease predominantly affecting males. Natural history models capture the full pathway under current care and combine with estimates of new interventions’ effects to assess cost‐effectiveness by health technology decision‐makers. These require mortality throughout patient's lifetime, but rare datasets typically contain relatively few patients short follow‐ups. Alternative (published) sources data may...
The aim of this study was to pool multiple data sets build a patient-centric, data-informed, natural history model (NHM) for Duchenne muscular dystrophy (DMD) estimate disease trajectory across patient lifetime under current standard care in future economic evaluations. conducted as part Project HERCULES, multi-stakeholder collaboration develop tools support health technology assessments new treatments DMD. Health states were informed by review NHMs DMD and input from clinicians, patients...
Abstract Background Patient and public involvement (PPI) ensures that research is designed conducted in a manner most beneficial to the individuals whom it will impact. It has an undisputed place applied required by many funding bodies. However, PPI statistical methodology more challenging work needed identify where how patients can meaningfully input this area. Methods A descriptive cross-sectional study was using online questionnaire, which asked methodologists about themselves their...
Patient and Public Involvement Engagement (PPIE) is important to all aspects of health research. However, there are few examples successful PPIE in statistical methodology One the reasons for this relates challenges identification individuals interested research projects, ambiguities over importance these projects.This project was conducted between August 2022 2023. The aim report process development an accessible animation describe what projects. For this, we combined storyboarding...
<title>Abstract</title> <italic><bold>Background</bold></italic> The aim of this study was to build a natural history model (NHM) for Duchenne muscular dystrophy (DMD) provide new insights into the disease and inform economic evaluations therapies. conducted as part Project HERCULES, multi-stakeholder collaboration develop tools support health technology assessment treatments DMD. <italic><bold>Methods</bold></italic> Health states were identified based on review NHMs input from clinicians,...
Abstract Background: Patient and Public Involvement (PPI) is important to all aspects of health research. However, there are few examples successful PPI in statistical methodology One the reasons for this relates challenges identification individuals interested methods, ambiguities over importance these projects. Methods: This project was conducted between August 2022 2023. The aim report process development an accessible animation describe what methods-based For this, we combined...
Multi‐state survival models are used to represent the natural history of a disease, forming basis health technology assessment comparing novel treatment current practice. Constructing such for rare diseases is problematic, since evidence sources typically much sparser and more heterogeneous. This simulation study investigated different one‐stage two‐stage approaches meta‐analyzing individual patient data (IPD) in multi‐state setting when number size studies being meta‐analyzed small. The...
Multi-state survival analysis considers several potential events of interest along a disease pathway. Such analyses are crucial to model complex patient trajectories and increasingly being used in epidemiological health economic settings. models often make the Markov assumption, whereby an individual's future trajectory is dependent only upon their present state, not past. In reality, there may be transitional dependence either previous and/or more than one timescale, for example time since...