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Understanding heart failure in Duchenne and Becker and developing an improved gene therapy
1 year agoHeart failure is a serious and common complication of Duchenne and Becker muscular dystrophy....
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Identifying new genetic causes of limb girdle muscular dystrophy
1 year agoDr Yalda Jamshidi and her team at St George’s University of London will use the latest genetic techniques to identify new causes of limb girdle muscular dystrophy (LGMD)....
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Improving the delivery of molecular patches for spinal muscular atrophy
1 year agoDr Suzan Hammond from the MDUK Oxford Neuromuscular Centre is developing molecular patches for spinal muscular atrophy (SMA) that can cross from the blood into the central nervous system....
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Screening for molecules that alter myosin function
1 year agoDr Arianna Fornili and her PhD student at Queen Mary University of London are searching for molecules that alter the function of a protein called myosin. This could lead to the development of potential treatments for nemaline myopathy.
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Developing new delivery methods for microdystrophin gene therapy
1 year agoProfessor Matthew Wood and his team at the MDUK Oxford Neuromuscular Centre are investigating new delivery methods for gene therapy....
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Investigating the incidence and prevalence of neuromuscular conditions in the UK
1 year agoDr Iain Carey and his team at St George’s University of London will use national health research databases to estimate the number of newly diagnosed cases of neuromuscular conditions (incidence) and the number of people currently living with a neuromuscu...
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Investigating the optimal settings for a cough assist device
1 year agoDr Patrick Murphy and his team, including research fellow Neeraj Shah, at Guy’s and St Thomas’ Hospital are using an artificial lung to identify the most optimal settings for a cough assist device....
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Using mini-muscles to test genome editing for laminopathies
1 year agoDr Francesco Saverio Tedesco and his PhD student at University College London will test genome editing tools in 3D ‘mini-muscles’ that model laminopathies....
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Investigating the role of INPP5K in congenital muscular dystrophy
2 years agoDr Laura Swan at the University of Liverpool will investigate why mutations in an enzyme called INPP5K cause a congenital muscular dystrophy. This will improve our understanding of the condition, which is key to developing potential treatments.
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Could an existing drug be a treatment for spinal muscular atrophy?
2 years agoDr Melissa Bowerman and her PhD student from Keele University will be investigating whether an already approved drug could be beneficial for spinal muscular atrophy (SMA).
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