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Dr Linda Popplewell and her team at Royal Holloway University will develop an antibody that targets muscle scarring (fibrosis) and test it in cell and mouse models with Duchenne muscular dystrophy. Fibrosis is a common feature of muscular dystrophies, so this research could lead to a potential treatment for many people with muscular dystrophies.
Dr Linda Popplewell and her team at Royal Holloway University will develop an antibody that targets muscle scarring (fibrosis) and test it in cell and mouse models with Duchenne muscular dystrophy. Fibrosis is a common feature of muscular dystrophies, so this research could lead to a potential treatment for many people with muscular dystrophies.
Dr Linda Popplewell and her team at Royal Holloway University will develop an antibody that targets muscle scarring (fibrosis) and test it in cell and mouse models with Duchenne muscular dystrophy. Fibrosis is a common feature of muscular dystrophies, so this research could lead to a potential treatment for many people with muscular dystrophies.
Dr Federica Montanaro at University College London (UCL) is investigating the impact that exon skipping has on the function of the dystrophin protein. The findings of this project will be important for designing future molecular patches for people with Duchenne muscular dystrophy.
Dr Federica Montanaro at University College London (UCL) is investigating the impact that exon skipping has on the function of the dystrophin protein. The findings of this project will be important for designing future molecular patches for people with Duchenne muscular dystrophy.
Dr Federica Montanaro at University College London (UCL) is investigating the impact that exon skipping has on the function of the dystrophin protein. The findings of this project will be important for designing future molecular patches for people with Duchenne muscular dystrophy.
Dr Shuko Joseph at the University of Glasgow has been awarded a clinical academic training fellowship to study bone health and how it changes in boys with Duchenne muscular dystrophy. This fellowship will help to increase research and clinical care capacity for Duchenne muscular dystrophy in Scotland.
Dr Shuko Joseph at the University of Glasgow has been awarded a clinical academic training fellowship to study bone health and how it changes in boys with Duchenne muscular dystrophy. This fellowship will help to increase research and clinical care capacity for Duchenne muscular dystrophy in Scotland.
Dr Shuko Joseph at the University of Glasgow has been awarded a clinical academic training fellowship to study bone health and how it changes in boys with Duchenne muscular dystrophy. This fellowship will help to increase research and clinical care capacity for Duchenne muscular dystrophy in Scotland.
In this project, Professor Dame Kay Davies and her team will continue their search for new compounds that can increase levels of a protein called utrophin. Utrophin is similar to dystrophin and found in small amounts in adult muscle. Increasing its levels might compensate for the lack of dystrophin seen in people with Duchenne muscular dystrophy. Next generation molecules, which have improved drug-like qualities or act through a different mechanism compared to older compounds, are needed to ensure more effective treatments are developed. This approach has the benefit of being applicable to all people with Duchenne or Becker muscular dystrophy, whatever their mutation. This work is funded by the UtroDMD Alliance, of which Muscular Dystrophy UK is a member.
In this project, Professor Dame Kay Davies and her team will continue their search for new compounds that can increase levels of a protein called utrophin. Utrophin is similar to dystrophin and found in small amounts in adult muscle. Increasing its levels might compensate for the lack of dystrophin seen in people with Duchenne muscular dystrophy. Next generation molecules, which have improved drug-like qualities or act through a different mechanism compared to older compounds, are needed to ensure more effective treatments are developed. This approach has the benefit of being applicable to all people with Duchenne or Becker muscular dystrophy, whatever their mutation. This work is funded by the UtroDMD Alliance, of which Muscular Dystrophy UK is a member.
In this project, Professor Dame Kay Davies and her team will continue their search for new compounds that can increase levels of a protein called utrophin. Utrophin is similar to dystrophin and found in small amounts in adult muscle. Increasing its levels might compensate for the lack of dystrophin seen in people with Duchenne muscular dystrophy. Next generation molecules, which have improved drug-like qualities or act through a different mechanism compared to older compounds, are needed to ensure more effective treatments are developed. This approach has the benefit of being applicable to all people with Duchenne or Becker muscular dystrophy, whatever their mutation. This work is funded by the UtroDMD Alliance, of which Muscular Dystrophy UK is a member.
In this project Professor Francesco Muntoni and Dr Francesco Conti are aiming to develop a new gene therapy approach called genome editing to repair mutations in the dystrophin gene caused by duplication of a sequence of DNA. Approximately 10 to 15 percent of people with Duchenne muscular dystrophy have this type of mutation. If successful, this technique could permanently correct the underlying genetic cause of the condition, meaning the therapeutic agent should not need to be administered multiple times.
In this project Professor Francesco Muntoni and Dr Francesco Conti are aiming to develop a new gene therapy approach called genome editing to repair mutations in the dystrophin gene caused by duplication of a sequence of DNA. Approximately 10 to 15 percent of people with Duchenne muscular dystrophy have this type of mutation. If successful, this technique could permanently correct the underlying genetic cause of the condition, meaning the therapeutic agent should not need to be administered multiple times.
In this project Professor Francesco Muntoni and Dr Francesco Conti are aiming to develop a new gene therapy approach called genome editing to repair mutations in the dystrophin gene caused by duplication of a sequence of DNA. Approximately 10 to 15 percent of people with Duchenne muscular dystrophy have this type of mutation. If successful, this technique could permanently correct the underlying genetic cause of the condition, meaning the therapeutic agent should not need to be administered multiple times.

Campaign Autumn 2019

We hope you enjoy reading your Autumn edition of Campaign, the newsletter that keeps you connected with the work you help fund.
We hope you enjoy reading your Autumn edition of Campaign, the newsletter that keeps you connected with the work you help fund.
Professor Henry Houlden and Professor Mary Reilly at University College London (UCL) are investigating new genetic changes that lead to early-onset neuropathies. This will help give a better understanding of what is going wrong in these conditions and will help direct future research into potential treatments.
Professor Henry Houlden and Professor Mary Reilly at University College London (UCL) are investigating new genetic changes that lead to early-onset neuropathies. This will help give a better understanding of what is going wrong in these conditions and will help direct future research into potential treatments.
Professor Henry Houlden and Professor Mary Reilly at University College London (UCL) are investigating new genetic changes that lead to early-onset neuropathies. This will help give a better understanding of what is going wrong in these conditions and will help direct future research into potential treatments.
Dr Spinazzola and colleagues at University College London (UCL) and Newcastle University are carrying out a pilot study testing a potential treatment in people with mitochondrial disease.
Dr Spinazzola and colleagues at University College London (UCL) and Newcastle University are carrying out a pilot study testing a potential treatment in people with mitochondrial disease.
Dr Spinazzola and colleagues at University College London (UCL) and Newcastle University are carrying out a pilot study testing a potential treatment in people with mitochondrial disease.

Moving up with mito: Jack's story

During Mitochondrial Disease Awareness Week (16-22 September), we’re featuring Jack Blackburn, a 19-year-old with this condition. Jack proved himself an excellent office worker alongside Trailblazers, our young disabled campaigning group, during a month’s Moving Up work experience placement in our London office. 
During Mitochondrial Disease Awareness Week (16-22 September), we’re featuring Jack Blackburn, a 19-year-old with this condition. Jack proved himself an excellent office worker alongside Trailblazers, our young disabled campaigning group, during a month’s Moving Up work experience placement in our London office. 

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Date Published:
16 September 2019
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