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UNITE-DMD - UK gene therapy trial

In March 2017, Action Duchenne announced our funding of “UNITE-DMD”, one of the first gene therapy trials for Duchenne muscular dystrophy here in the UK.

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Splicing efficiency project

Enhancement of splicing efficiency between trans‐spliced DMD AAV vectors for the production of full‐length fully functional dystrophin protein.

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Genome editing surgery

Development of genome editing surgery with applicability to all living with Duchenne – Royal Holloway University

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AAV-U7

Benefit evaluation of AAV-U7 treatment combined with corticosteroids for Duchenne muscular dystrophy – Institute of Myology

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SKIP-NMD (exon 53 programme)

In late 2012, we awarded a small grant to be involved in the SKIP-NMD programme. SKIP-NMD is an EU FP7 funded collaborative grant involving 10 partners from Europe and the USA, whose aim is to restore dystrophin production in a subset of DMD boys.

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PepGen

Hilary Hoskin legacy funds exon skipping research project

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ScOT-DMD bone health study

Dr Shuko Joseph has been awarded the funds for a three-year project to better understand bone deterioration in the 2,500 children and young people living with Duchenne muscular dystrophy in the UK.

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Set-up fund for adult Duchenne trials

To enable Ros Quinlivan and her team at the University College London to fund researchers so that trials can continue to run for adults.

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Validating outcome measures

A novel device called ActiMyo® has been proposed for its use as an outcome measure in ambulant and non-ambulant Duchenne trials.

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Repurposed Cancer Therapeutics

Repurposed Cancer Therapeutics as Treatments for DMD, the project will be led by Professor Steve Winder in the Department of Biomedical Science at the University of Sheffield.

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