Search icon Open mobile navigation
Dr 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.
Dr 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.
Dr 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.

Response to second negative opinion for Duchenne muscular dystrophy treatment Exondys 51

Submitted by Anonymous (not verified) on Fri, 09/21/2018 - 00:00
Exondys 51 is not a cure, but it has the potential to slow down the progression of this devastating condition, which would buy families valuable time and keep children walking for longer.
Exondys 51 is not a cure, but it has the potential to slow down the progression of this devastating condition, which would buy families valuable time and keep children walking for longer.
Dr Lunn and his team will test a non-invasive magnetic resonance imaging (MRI) method for its ability to be used as a diagnostic tool for people with inflammatory neuropathy.
Dr Lunn and his team will test a non-invasive magnetic resonance imaging (MRI) method for its ability to be used as a diagnostic tool for people with inflammatory neuropathy.
Dr Lunn and his team will test a non-invasive magnetic resonance imaging (MRI) method for its ability to be used as a diagnostic tool for people with inflammatory neuropathy.
Professor Francesco Muntoni at University College London (UCL) will identify new genes associated with congenital muscular dystrophy or congenital myopathy and investigate their function. This will help to speed up the diagnosis of families with these conditions.
Professor Francesco Muntoni at University College London (UCL) will identify new genes associated with congenital muscular dystrophy or congenital myopathy and investigate their function. This will help to speed up the diagnosis of families with these conditions.
Professor Francesco Muntoni at University College London (UCL) will identify new genes associated with congenital muscular dystrophy or congenital myopathy and investigate their function. This will help to speed up the diagnosis of families with these conditions.

Statement on Spinraza access raised at PMQs by Mary Glindon

Submitted by Anonymous (not verified) on Thu, 09/13/2018 - 00:00

Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK, said:

NICE, NHS England and Biogen must urgently come together to find a solution and put an urgent, temporary scheme in place for Spinraza before any more children’s lives are lost. Without access to this life-changing treatment infants will irreversibly lose the ability to walk, crawl and swallow, while babies with the most severe form of the condition are likely to die before they reach their second birthday.

Professor Jenny Morgan and her team aim to better understand the mechanisms of muscle fibre death in Duchenne muscular dystrophy and investigate whether this process could be manipulated with therapeutic benefit.
Professor Jenny Morgan and her team aim to better understand the mechanisms of muscle fibre death in Duchenne muscular dystrophy and investigate whether this process could be manipulated with therapeutic benefit.
Professor Jenny Morgan and her team aim to better understand the mechanisms of muscle fibre death in Duchenne muscular dystrophy and investigate whether this process could be manipulated with therapeutic benefit.
Professor Henry Houlden at University College London is investigating whether an enzyme called Sirt1 has a role in muscle repair in Duchenne muscular dystrophy. Findings from this study will determine if targeting Sirt1 could help to treat Duchenne.
Professor Henry Houlden at University College London is investigating whether an enzyme called Sirt1 has a role in muscle repair in Duchenne muscular dystrophy. Findings from this study will determine if targeting Sirt1 could help to treat Duchenne.
Professor Henry Houlden at University College London is investigating whether an enzyme called Sirt1 has a role in muscle repair in Duchenne muscular dystrophy. Findings from this study will determine if targeting Sirt1 could help to treat Duchenne.
Heart failure is a serious complication of Duchenne muscular dystrophy and Becker muscular dystrophy and current drug treatments are inadequate. The student working on this PhD studentship at UCL, supervised by Professor Jenny Morgan and Dr Federica Montanaro, will study heart function at a molecular level to gain useful information that will inform the development of future gene therapy.
Heart failure is a serious complication of Duchenne muscular dystrophy and Becker muscular dystrophy and current drug treatments are inadequate. The student working on this PhD studentship at UCL, supervised by Professor Jenny Morgan and Dr Federica Montanaro, will study heart function at a molecular level to gain useful information that will inform the development of future gene therapy.
Heart failure is a serious complication of Duchenne muscular dystrophy and Becker muscular dystrophy and current drug treatments are inadequate. The student working on this PhD studentship at UCL, supervised by Professor Jenny Morgan and Dr Federica Montanaro, will study heart function at a molecular level to gain useful information that will inform the development of future gene therapy.
Dr Ros Quinlivan at University College London (UCL) is aiming to drive the development of the adult North Star Network. This project will establish a set of standardised outcome measures that will help to improve overall care for adults with Duchenne muscular dystrophy.
Dr Ros Quinlivan at University College London (UCL) is aiming to drive the development of the adult North Star Network. This project will establish a set of standardised outcome measures that will help to improve overall care for adults with Duchenne muscular dystrophy.
Dr Ros Quinlivan at University College London (UCL) is aiming to drive the development of the adult North Star Network. This project will establish a set of standardised outcome measures that will help to improve overall care for adults with Duchenne muscular dystrophy.
Dr Gonzalo Blanco at the University of York will investigate the function of a muscle protein called KY. This research will improve our understanding of muscle biology and how this is affected in a type of congenital myopathy.
Dr Gonzalo Blanco at the University of York will investigate the function of a muscle protein called KY. This research will improve our understanding of muscle biology and how this is affected in a type of congenital myopathy.
Dr Gonzalo Blanco at the University of York will investigate the function of a muscle protein called KY. This research will improve our understanding of muscle biology and how this is affected in a type of congenital myopathy.
Subscribe to