making muscle wasting history

Will the Governments Health reforms improve Standards of Care for those living with Duchenne?

Nick Catlin CEO of Action Duchenne introduces the discussion on the recent Government proposals to reform Health and the impact of recent cuts in services. Kate Bushby from Newcastle explains the internationally agreed standards of care for Duchenne. Adnan Manzur describes the the Great Ormond Street model of a Centres of Expertise. Stuart Wickison who is living with Duchenne tells us all about the problems of accessing current services.

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Will the Governments Health reforms improve Standards of Care for those living with Duchenne?

Kate Bushbys presentation on Standards of care for Duchenne during this discussion

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Will the Governments Health reforms improve Standards of Care for those living with Duchenne?

Adnan Manzurs presentation during this discussion about Great Ormond Street's model of Care.

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Caring for the Heart

John Bourke from Newcastle presents important information on managing the heart complications for Duchenne.

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New methods for detailed genetic analysis

Steve Abbs Guys Hospital discusses improved ways of analysing the dystrophin gene variation

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Autonomy - the Transition to Adulthood 14 - 19 years

Emily Ballard Guys and St Thomas' London and Gordon McClurg Care Management Services discuss here the issues that need to be addressed during transition to adulthood.

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Respiratory Care for Duchenne

Nick Hart from the Lane Fox Unit London describes how crucial respiratory care is for older people living with Duchenne.

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Bone protection for children with DMD treated with corticosteroids

Ros Quinliven Great Ormond Street Hospital discuss one of the side effects of long term steroid use and offers ways of helping to provide better bone protection.

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Duchenne, ADHD, Dyslexia and Autism

Veronica Hinton Columbia University USA and James Poysky USA discuss the increased risks that young people living with Duchenne have of specific learning and behaviour problems.

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Pilot Trials Now

George Vella from Charley's Fund in the USA gives an update on Pilot Trials Now that aims to fast track trials in Duchenne patients of promising drugs already used for other conditions. Current trials underway include Sildenifil and IGF1.

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Melatonin

Dario Acuna-Castroviejo Univerity of Granada Spain presents his teams clinical trial using melatonin as a potential treatment for Duchenne

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Laminin 111

Richard Cloud and Brad Hodges from Prothelia USA discuss their novel approach to use Laminin 111 to help to stabilise the muscle cell membrane.

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Human Genome at 10: Patient Trials for Duchenne

Professor Dame Kay Davies University of Oxford reviews the progress that has been made in developing new drugs and treatments for Duchenne.

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Exon skipping - improving delivery and efficiency

Steve Wilton from University of Western Australia Chairs a discussion on emerging ways to improve exon skipping and delivery into cells. Mike Gait from University of Cambridge explains how his collaboration with Matthew wood at University of Oxford are progressing with improved peptide AO design. Aurelie Goyenvalle from University of Oxford describes improvements in U7/AAV delivery for exon skipping and multiple skipping.

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Exon skipping - improving delivery and efficiency

Mike Gait from University of Cambridge explains how his collaboration with Matthew Wood at University of Oxford is progressing with improved peptide AO design. With Steve Wilton and Aurelie Goyenvalle.

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Exon skipping - improving delivery and efficiency

Aurelie Goyenvalle from University of Oxford describes improvements in U7/AAV delivery for exon skipping and multiple skipping. With Mike Gait and Steve Wilton.

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How will the regulatory framework help to support the development of new gene therapies for Duchenne?

Pavel Balabanov from the European Medicines Agency describes the role played by regulatory authorities in supporting new drug development. Pavel comes to some useful and interesting conclusions on the role that the EMA can play in supporting principal investigators and biotech companies in bringing down some of the barriers.

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Improving Clinical trial design and outcomes

Kate Bushby explains the need to improve design for Duchenne clinical trials. There is also an urgent need to agree outcome indicators of clinical improvement and develop sensitive biomarkers that can show drug efficacy.

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Duchenne Research in China

Haifang Yin has recently set up a new research lab in China in partnership with University of Oxford. Haifang explains how research is moving ahead in China and the Duchenne projects that she will be leading.

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Rare Diseases and personalised medicines

Padraig Wright who is vice President of clinical neuroscience at Glaxo Smith Kline discusses the companies development of a new rare disease unit and also GSK's Duchenne gene therapy clinical trials.

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The development of Idebenone (Catena) for the treatment of DMD

Dr Raffaelle Robino speaks about the upcoming clinical trials for Santhera's Catena as a treatment for Duchenne

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