Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 20 of 27 for “"Disease-modifying therapies"”.
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Assessing Disease Modifying Therapies in Autosomal Dominant Polycystic Kidney Disease
… growth in autosomal dominant polycystic kidney disease (ADPKD) and is a key therapeutic target. Evaluation of high water intake (HWI) as an alternative to pharmacological vasopressin blockade is supported by patients. However the feasibility, safety and adherence-promoting strategies required to …
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Designing nanocarriers to penetrate cartilage and improve delivery of biologic drugs for osteoarthritis
Osteoarthritis is a debilitating joint disease that affects over 30 million people and has no disease-modifying therapies. The current standard of care for the disease is merely palliative until joint replacement is necessary. Disease-modifying osteoarthritis drugs have been tested in the clinic, …
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Developing Sphingosine-1-Phosphate (Spns2) Inhibitors for the Treatment of Multiple Sclerosis
Autoimmune diseases are caused when a person's immune system attacks its own healthy cells. In a person with multiple sclerosis, their immune system becomes sensitized to the myelin sheath that covers their neurons in the central nervous system. This results in the degradation of the myelin sheath …
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Identification and evaluation of biomarkers for Huntington’s disease
Huntington’s disease (HD) is a devastating, incurable inherited neurodegenerative disorder that commonly affects adults in mid-life. Despite encouraging results from in vitro and animal trials, disease-modifying therapeutic trials in HD are limited by a lack of tools to track disease progression. …
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The independent and combined effects of rapamycin and metformin on naturally occurring osteoarthritis
Osteoarthritis (OA) is among the top 10 diseases limiting human healthspan, and no disease modifying therapies currently exist. OA is a degenerative disease of the whole joint, characterized by articular cartilage loss, subchondral bone sclerosis, meniscal calcification, synovitis, and skeletal …
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The role of Hsp60 in Amyloid beta toxicity: Relevance to Alzheimer’s disease
Alzheimer’s disease (AD) is a devastating neurodegenerative disorder leading to dementia, affecting millions of individuals worldwide. Currently, there are no successful disease-modifying therapies, due to the complexity of biochemical alterations and risk factors leading to AD. One of the earliest …
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The role of Hsp60 in Amyloid beta toxicity: Relevance to Alzheimer’s disease
Alzheimer’s disease (AD) is a devastating neurodegenerative disorder leading to dementia, affecting millions of individuals worldwide. Currently, there are no successful disease-modifying therapies, due to the complexity of biochemical alterations and risk factors leading to AD. One of the earliest …
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Mechanisms of Action in Motivational Interviewing
Despite the demonstrated efficacy of disease modifying therapies (DMT), many MS patients (~ 40%) stop using DMT within 3 years of starting. A cross-over randomized controlled trial demonstrated that a theoretically based motivational interviewing- cognitive behavior therapy (MI-CBT) intervention …
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Machine Learning Methods for Personalized Treatment Response Characterization Using Clinical Care Brain MRI and Non-Imaging Data in Multiple Sclerosis
… (MS) is a common and disabling neurological disease with increasing cost. Most people begin MS with a relapsing-remitting form (RRMS) but disease trajectory differs by person. There are over 20 low- and high-efficacy disease modifying therapies (DMTs) for people with RRMS; however, many of …
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Modulating System xc- Activity As A Treatment For Epilepsy
… to 1-in-3 epileptic patients. Additionally, no disease modifying therapies currently exist to treat the underlying pathological processes involved in epileptogenesis. The overarching goal of this project is to further characterize the role astrocytes play in epileptogenesis, in hopes of …
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SMN DEFICIENCY INDUCES NEUROACTIVE AMINOACIDS DYSMETABOLISM: BRIDGING INSIGHTS FROM PRECLINICAL MODELS TO CLINICAL EVIDENCE IN SMA PATIENTS
… the SMN1 gene and reduced SMN protein. Although disease-modifying therapies such as Nusinersen, Risdiplam, and Onasemnogene abeparvovec have markedly improved survival and motor outcomes, they are not curative, and many patients continue to exhibit residual weakness, metabolic instability, and …
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Examinations into the Calcium Hypothesis of Alzheimer's Disease
Alzheimer's disease (AD) is devastating to the patient, their family and friends, and represents a significant fiscal burden to our society. Currently available therapeutics provide only mild symptomatic relief and do not alter the course of the disease. Developing the next generation of disease …
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Imaging and therapy in multiple sclerosis
… (RR) MS for which numerous immunomodulatory disease-modifying therapies (DMTs) are licensed. However, after approximately 20 years most convert to secondary progressive (SP) MS where immunomodulatory therapies have little if any effect. Whether DMTs delay or prevent this transition remains …
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Regulation of autophagosome formation and maturation by neurodegeneration-associated proteins
… organelles associated with neurodegenerative diseases. Autophagy cargoes are captured into double-membraned autophagosomes, which form as outgrowths from the Ras-related protein 11A (RAB11A)-positive recycling endosomes. Autophagosomes are closed by the endosomal sorting complex required for …
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Genetic characterisation of the Drosophila Mitochondrial Calcium Uniporter in physiological and neurodegenerative contexts
Neurodegenerative conditions such as Alzheimer’s disease (AD) and Parkinson’s disease (PD) are a growing medical and social burden for which no disease-modifying therapies exist, necessitating greater understanding of their underlying pathobiology. Mitochondrial health and calcium signalling have …
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Investigating the role of cellular communication network factor 3 (CCN3) in central nervous system myelination and remyelination
Multiple sclerosis (MS) is a demyelinating disease of the central nervous system (CNS) characterised by immune-mediated destruction of myelin-producing oligodendrocytes. While there is a plethora of disease-modifying therapies to reduce relapses, there are currently no available treatments to boost …
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The feasibility of a patient informed, racially targeted home-based exercise program for black individuals with multiple sclerosis
… with MS may experience a more aggressive disease course, have a poorer prognosis with disease modifying therapies and have increased odds of physical comorbidities than their white counterparts. There is substantial evidence that suggests exercise training to be a promising approach for …
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Designing a Quantitative Videofluoroscopic Analysis Approach in Infants with Spinal Muscular Atrophy Type 1
… deterioration in infants. Recent FDA approval of disease-modifying therapies have led to improvements in survival and motor function; however, their effects on bulbar physiology remains unclear. In this study, we aimed to establish a videofluoroscopic swallow study (VFSS) analysis method that …
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An investigation of novel therapeutic strategies for the treatment of multiple sclerosis and autoimmunity
Autoimmune disease is characterised by the development of abnormal immune responses directed against antigenic components of the host i.e. self-antigen. Multiple sclerosis (MS) is a chronic autoimmune, inflammatory disease directed against an unknown antigen in the central nervous system. This …
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The role of tau and neuroinflammation in Progressive Supranuclear Palsy and Alzheimer's Disease
… Supranuclear Palsy (PSP) and Alzheimer’s disease (AD) ‘in vivo’ and to validate it using post mortem data is critical to develop reliable biomarkers for these disorders, and for tracking the effects of clinical trials using disease-modifying therapies. The main aim of my PhD was therefore …
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