BBC puts spotlight on access to potential Duchenne treatments

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The BBC is featuring access to potential treatments for Duchenne muscular dystrophy today between midday and 1pm on BBC Radio 5 Live which will include contributions from the Halpin family, the Hill family and the Muscular Dystrophy Campaign.

This follows on from the recent launch of the Muscular Dystrophy Campaign's Fast Forward campaign. The calls to action to speed up access to specialist treatments include:

  • Ensuring that cutting-edge, high-cost potential treatments for types of muscle-wasting condition are not held up due to lack of funding
  • Making sure that treatments for muscle-wasting conditions are assessed separately from cheaper drugs for more common conditions, and therefore not denied on grounds of high-cost
  • Delivering high-quality, consistent standards of care across the UK
  • Increased support for clinical trials infrastructure, such as patient registries and databases

The feature follows last week's announcement by the European Commission granting conditional approval for Translarna (formerly known as ataluren), a drug designed to treat the 10 - 15% of boys and young men with Duchenne muscular dystrophy caused by a 'nonsense mutation'.

The Muscular Dystrophy Campaign is working with the NHS through the Bridging the Gap project to ensure sufficient support is provided to develop multi-disciplinary teams at centres of excellence. Consistently high standards of care would also mean patients' conditions are managed to the best possible level, thereby enabling them to take part in trials for drugs to treat muscle-wasting conditions.

Links and further information

Learn more about Duchenne muscular dystrophy

Read about the research we are funding into Duchenne muscular dystrophy

We are here for all people in the UK affected by Duchenne muscular dystrophy. If you need information, support or advice please contact us on 0800 652 6352 (freephone) or 

It is only through your contributions that we can continue to fund the vital work that takes us closer to finding treatments and cures for muscle-wasting conditions. Donate now and help change the lives of thousands of people living with muscular dystrophy and related neuromuscular conditions.

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