making muscle wasting history

Medicines For Duchenne

Action Duchenne was established to find a cure or more effective medicines to treat Duchenne Muscular Dystrophy.

Since 2001 we have funded key research projects and supported clinical trials that are making real progress towards our key aim.

Funding for new Exon Skipping medicines

Action Duchenne has been a key partner in the UK MDEX consortium that has been developing new antisense oligonucleotides that are able to produce new dystrophin proteins in muscle cells that we hope will eventually slow the very severe muscle wasting found in Duchenne.

In 2003 we worked with key UK clinical and research groups to win £2.2m of Department of Health and Government funding for the MDEX consortium to conduct pre clinical studies and establish two groundbreaking UK clinical trials.

Since 2003 Action Duchenne has directly funded further research in exon skipping in the UK and Australia with £300,000 of further grants to teams at Imperial College London, Oxford University, Cambridge University and University of Western Australia.

$1.2m for Exon Skipping research with AVI Biopharma

In 2009 Action Duchenne established a key research programme with AVI Biopharma in the United States. The aim of the project was to

a) Fund work with Regulatory authorities such as EMEA, MHRA to enable Exon Skipping drug sequences to be seen as a platform medicine

b) To put in place three new clinical trial coordinator posts to establish further UK and EU clinical trial sites

c) Further pre clinical development of antisense oligonucleotide sequences to enable more young people living with Duchenne to be treated by exon skipping drugs.

Utrophin Upregulation

In 2008/9 Action Duchenne funded Summit plc for £175K to support further development of their utrophin upregulation programme. Summit have recently sold on their key compound SUMC1100 to Biomarin in the USA who are proposing to take this into clinical trial in 2010.

Stem Cells

Action Duchenne has also funded research at UCL to investigate ways to develop stem cells to treat Duchenne.

DMD Registry

Action Duchenne has established and continues to fund a Registry of Duchenne patients that collects vital gene variation information to help recruitment for genetic clinical trials.

Donate to our Medicines for Duchenne campaign for 2010