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DMD research

Taurine Project - an update on progress

4th March 2020

Duchenne UK is pleased to be able to update the community with the latest on a project it funds into the supplement taurine.

Many parents give their children supplements – some of which are very expensive – but there’s little evidence they’re safe or effective.

Taurine is an amino acid found in the tissue of all animals, which can be bought as a health food supplement. It’s cheap, can be taken orally and it’s understood to be relatively safe for humans.

There’s strong pre-clinical data in a DMD (mdx) mouse model to suggest that it improves muscle strength, reduces inflammation and protects against muscle wasting.  That’s why we’re investing in research into it – if proved to be effective it could be an exciting treatment for Duchenne Muscular Dystrophy.

In September 2018, the charity made a grant of £273,468 to Dr Peter Arthur and his team at the University of Western Australia (UWA) who are investigating the use of taurine for DMD.

Currently they’re looking at its effects in a new rat model, developed by French collaborators, which has a more severe form of muscle damage.

The researchers have to find a way to measure taurine’s effectiveness. They’re trying to identify molecules in the blood or in urine (biomarkers) which show that the substance is working in the way they want it to in the muscles.

Because Taurine is believed to have an anti-oxidant effect which reduces inflammation in muscle, the team are focussing on oxidative stress measures in the blood and urine.

Dr Arthur tells us that the team has identified a promising oxidative stress measure in blood called oxidised albumin. They’ve found the levels of oxidised albumin are raised in the blood of mice with DMD, but that these levels decrease when they’re treated with taurine.

In addition, Jessica Terrill, of the UWA team has found a second oxidative stress biomarker which is present in urine.

The team are also looking at how it is best to introduce taurine into the body and in what quantities. If taurine is shown to work in rats it will need further testing in humans.

The research is continuing – thanks to our grant. We’ll keep you updated with the progress of the team from UWA and their French collaborators.


NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy.

We are investing millions of pounds in research right now to bring treatments and a cure to help this generation. Duchenne UK is the largest funder of DMD research in the UK. We are also committed to accelerating the pace of research. 90p in every £1 raised is committed to research.

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

We need your help, because we need to keep funding promising new research.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

  • Direct Debit – Duchenne Direct
  • Individual Donation – Donate
  • If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
  • Take part in one of our fundraising events – Events
  • Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information visit www.duchenneuk.org

Published on 3 April 2020

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