Skip to content

Important announcement for adults with Duchenne muscular dystrophy

18 December 2020

Muscular Dystrophy UK is delighted to announce the successful completion of two major projects we funded to improve care and support for adults living with Duchenne muscular dystrophy (DMD).

These include the first ever national Standards of Care for adults with DMD and the expansion of the North Star natural history database.

Adult Duchenne Standards of Care

While best practice care considerations for the care of children living with Duchenne have been in place since 2010, there have been none for adults – until now.

In a pivotal moment, Professor Ros Quinlivan, Consultant in Neuromuscular Disorders at UCLH and lead of the Adult North Star Network, presented the first ever Standards of Care for Adults with Duchenne to the Word Duchenne Academy earlier this month.

The document includes vital guidance on:

  • Respiratory
  • General anaesthesia
  • Cardiology
  • Renal
  • Nutrition and bowel
  • Psychosocial
  • Palliative care

MDUK is pleased to announce that it has started important work tailoring a version of the care standards specifically for people living with Duchenne and their families. This resource alongside specific guidance for therapists will be made available next year.

The North Star Adult natural history database

An exciting new extension to the North Star natural history database – the Adult North Star database – has gone live.

It means, for the first time ever, the impact of Duchenne on adults is being recorded. This marks an important moment and opens up the possibility of adults accessing potential new treatments and clinical trials in the future.

MDUK also funded physiotherapy assessment (Performance of Upper Limb) equipment for every Adult North Star Network neuromuscular service across the UK as part of the project.

This will help specialist physios add accurate data to the natural history database.

What do these important developments mean for the UK’s Duchenne community?

  • We will be able to use the standards of care as a benchmark for improving good care in adults living with Duchenne across the UK
  • We will be able to work with NHS decision makers to highlight the need for increased investment in care
  • It will be easier for health professionals working with adults to share best practice with each other
  • The impact of Duchenne on adults will be recorded in a standardised way, making it possible to measure and monitor the impact of potential new treatments

Background to the Adult North Star Network

In 2016, MDUK funded a PhD project to drive forward the work of The Adult NorthStar Network to improve the quality of care for adults by:

  • Building a network of specialist health professionals who provide care for adults with Duchenne
  • Developing best practice holistic care standards and using these to improve equality of access to specialist support
  • Creating appropriate data capture forms for clinicians and physiotherapists working with adults with Duchenne
  • Expanding the North Star natural history database to ensure there is a record of the progression of Duchenne and measure the effectiveness of potential treatments

Achievements of the Adult North Star Network so far:

  • A national network of specialist health professionals working with adults with Duchenne
  • Completed standards of clinical care for adults with Duchenne (an equivalent care standard for therapists (physios, OTs) will be launched in 2021)
  • A live natural history database

Catherine Woodhead, Chief Executive of Muscular Dystrophy UK, said:

“I am extremely grateful to Professor Quinlivan and members of the Adult North Star Network for uniting to produce the first ever national best practice guidance for adults living with Duchenne.

“We are so pleased to have been able to support the development of these potentially game-changing resources.

“We will now work with clinicians, people living with Duchenne and NHS decision makers to secure equal access to best practice care for anyone living with Duchenne and other neuromuscular conditions in the UK.”

Professor Ros Quinlivan said:

“I am so pleased that thanks to the support of MDUK, the network has worked so well together and created the first comprehensive standards of care for adults with Duchenne anywhere in the world.

“For too long adult services for Duchenne have gone without the support and resources they need and the experience for young people moving from children’s to adult Duchenne care has been poor.

“With the standards of care and new Adult North Star database I hope we can continue to lead the way and improve care, support and research into potential new treatment that could benefit adults living with Duchenne.”

The Standards of Care are now being peer reviewed by international clinical experts in neuromuscular conditions and will be published online soon.

Vivek Gohil, who lives with Duchenne muscular dystrophy, knows all too well how important it is to have a national Standard of Care.

He said:

“I really noticed a change in the care I received when I moved from my children’s neuromuscular team into adult services. I know that this is not the case for everyone across the UK, but for me the change was really stark, with less time and access to a multi-disciplinary team who are experts in Duchenne.

“This all happened at a time when I felt very alone and my condition was really deteriorating and I actually needed more expert support. I’m really pleased that this new best practice guidance is now complete and hope we can level up care for adults with Duchenne and other neuromuscular conditions across the country.”

Join the Muscular Dystrophy UK community

Share your experience and find advice from others with similar conditions.