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ContactLimb girdle muscular dystrophy/Miyoshi myopathy/Duchenne muscular dystrophy, Prof K. Bushby
Group/Researcher: Prof K. Bushby
Prof Bushby will investigate whether the chemical Poloxamer 188 has the potential to be used as a therapy for muscular dystrophy.
Muscular dystrophies are a group of inherited diseases leading to muscle weakness and wasting. Over the last years, extensive research has been undertaken to understand how these disorders come about. One key theme emerging from this knowledge is the importance of an intact muscle cell membrane. All cells need a stable membrane, but skeletal muscle is special because the repeated cycles of contraction and relaxation apply extra stresses onto the cell membranes and create membrane breaches.
If the cell membrane is fragile to start with (as happens in Duchenne muscular dystrophy) or has a faulty mechanism for repairing those breaches (as it is the case with muscular dystrophies related to the protein dysferlin, limb girdle muscular dystrophy type 2B or Miyoshi Myopathy) the consequence is a progressive loss of muscle cells leading to muscular dystrophy.
Poloxamer is a drug that has the potential to repair damaged membranes and was found to have a protective effect on heart muscle cells after acute mechanical damage. This research will investigate if long-term application of Poloxamer will reduce the loss of muscle fibres in these types of muscular dystrophy and improve muscle function, and therefore might be a therapeutic drug for muscular dystrophy.
Value of Grant: Year 2 - £32,197
Prof Bushby will investigate whether the chemical Poloxamer 188 has the potential to be used as a therapy for muscular dystrophy.
Muscular dystrophies are a group of inherited diseases leading to muscle weakness and wasting. Over the last years, extensive research has been undertaken to understand how these disorders come about. One key theme emerging from this knowledge is the importance of an intact muscle cell membrane. All cells need a stable membrane, but skeletal muscle is special because the repeated cycles of contraction and relaxation apply extra stresses onto the cell membranes and create membrane breaches.
If the cell membrane is fragile to start with (as happens in Duchenne muscular dystrophy) or has a faulty mechanism for repairing those breaches (as it is the case with muscular dystrophies related to the protein dysferlin, limb girdle muscular dystrophy type 2B or Miyoshi Myopathy) the consequence is a progressive loss of muscle cells leading to muscular dystrophy.
Poloxamer is a drug that has the potential to repair damaged membranes and was found to have a protective effect on heart muscle cells after acute mechanical damage. This research will investigate if long-term application of Poloxamer will reduce the loss of muscle fibres in these types of muscular dystrophy and improve muscle function, and therefore might be a therapeutic drug for muscular dystrophy.
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