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Accelerating potential treatments for DMD through repurposing

Accelerating Potential Treatments for DMD: Researching a combination of existing medicines to treat Duchenne muscular dystrophy


Duchenne UK is pleased to announce a new research project to test existing medicines to see if they are effective in treating Duchenne muscular dystrophy. 

Duchenne UK is partnering with Professor Dominic Wells and the Royal Veterinary College, to investigate the possibility that a number of existing medicines prescribed for a variety of illnesses could be tested for efficacy in Duchenne muscular dystrophy. 

This exciting approach, known as repurposing, uses existing medicines which are already approved for use, and have been tested on humans; so information on their pharmacology, formulation and potential toxicity already exists.

This dramatically reduces the time it can take to get medicines into the clinic to be tested on patients.

Discovering and developing brand new treatments takes time and money. The translation of a promising new molecule into a drug approved for use in patients, can take anything up to 20 years.

Emily Crossley & Alex Johnson, co-founders of Duchenne UK said:

 “As part of our research strategy, we have been interested for some time in looking at the effects that repurposed drugs could have as a treatment option for Duchenne muscular dystrophy. We are delighted to have formed this partnership with Professor Wells, to push forward our understanding of these potential therapies as quickly as possible.”

There is an urgent need for safe effective treatments for Duchenne muscular dystrophy. 

It is hoped that a combination of drugs will be able to target the many effects in the muscle, caused by a lack of dystrophin.

Over a one year period, Professor Wells and his team will test six different drugs that are already approved for use in children, with conditions like cardiomyopathy and diabetes. 

Professor Wells will assess them in an Mdx mouse model of Duchenne to assess which combination offers the most promising avenue for treatment.

The optimal combination will be developed, with a view to conducting a human clinical trial.

Duchenne UK has invested £140,000 in this project, payable in 4 milestone dependent installments.  

Published on 25 May 2016

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