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Hereditary Motor Neuronopathies And Neuropathies: Mechanisms Of Neurodegeneration And Potential Modification.
Funder
National Health and Medical Research Council
Funding Amount
$104,664.00
Summary
The aim of the study is to investigate the pathophysiology of motor neuron degeneration in its various acquired and inherited forms. Nerve function will be determined by clinical and neurophysiological assessments in patients. The findings of the study will reveal further insights into the cause and progression of disorders of the motor neuron. This data may function to monitor disease progression, response to potential therapies and lead to the development of further therapeutic strategies.
Development Of A New Method Of Motor Unit Number Estimation For Use In Motor Neurone Disease
Funder
National Health and Medical Research Council
Funding Amount
$480,127.00
Summary
This project aims to help understand motor neurone disease, which is a severe disease that leads to paralysis and death. In motor neurone disease there is of death of the nerve cells that maks muscles move. We have developed a new method of measuring the number of motor nerve cells. We will use this to study the different types of motor neurone disease.
Flecainide In Amyotrophic Lateral Sclerosis - A Neuroprotective Strategy
Funder
National Health and Medical Research Council
Funding Amount
$593,275.00
Summary
This project will provide clinical trial information related to the potential neuroprotective properties of flecainide in motor neurone disease patients. A potential therapeutic response would provide impetus for a larger scale, multi-centre clinical trial. In addition to providing information about potential mechanisms of neurodegeneration and their treatment, new quantifiable measures will be further developed to objectively monitor MND patients in a clinical trials setting.
Characterization Of The FHL Protein Family In Striated Muscle
Funder
National Health and Medical Research Council
Funding Amount
$500,750.00
Summary
This grant examines the role of a family of muscle proteins, called FHL proteins, in skeletal and heart muscle. Inherited muscular disorders such as muscular dystrophy and myopathies, cause muscle weakness, which may be profound and lead to premature death due to respiratory muscle failure, or cause mild weakness later in life. The proteins which are defective in these muscular dystrophies are structural muscle proteins, which link and stabilize the contractile fibres in muscle and protect the m ....This grant examines the role of a family of muscle proteins, called FHL proteins, in skeletal and heart muscle. Inherited muscular disorders such as muscular dystrophy and myopathies, cause muscle weakness, which may be profound and lead to premature death due to respiratory muscle failure, or cause mild weakness later in life. The proteins which are defective in these muscular dystrophies are structural muscle proteins, which link and stabilize the contractile fibres in muscle and protect the muscle from the stresses and damage resulting from repeated muscular contraction. We have identified that the FHL proteins, which are the focus of this grant application, bind to and potentially regulate muscle proteins, which have been shown to cause forms of muscular dystrophy and cardiomyopathy. Examination of these interactions will provide insights into the biological mechanism of these muscle disorders. Furthermore, one of these proteins, FHL1 is significantly increased in hypertrophic cardiomyopathy, heart muscle thickening, a major cause of sudden cardiac death in young adults. We are creating transgenic mice, which make increased levels of FHL1 protein in their heart muscle, to determine whether increased FHL1, by itself is sufficient to promote heart muscle thickening. These studies should lead to further understanding of the development of diseases of heart and skeletal muscle, which may lead to novel treatments in the future.Read moreRead less