Celebrating 20 years: NorthStar a ‘shining light’ for people living with Duchenne muscular dystrophy
7 September 2024
We’re marking 20 years of the NorthStar programme, designed to gain a better understanding of Duchenne muscular dystrophy, and looking back at the achievements made over the last two decades.
A vision – then and now
Duchenne muscular dystrophy (DMD) is a severe and progressive muscle wasting condition, which mainly affects boys and men. Owing to the lack of the dystrophin protein, muscle fibres break down and are replaced by fibrous and or fatty tissue causing the muscle to weaken gradually. Boys will usually need a wheelchair by the age of about eight to 11 years.
Professor Francesco Muntoni, Paediatric Neurologist at Great Ormond Street Hospital, and Chief Investigator for NorthStar and Dr Adnan Manzur, consultant paediatrician at Great Ormond Street Hospital, and co-Principal Investigator for NorthStar, had a vision to learn about the natural history of DMD to improve standards of care and quality of life of boys living with the condition. Since it first began in 2004, we’ve invested more than £1.5 million in the NorthStar programme.
Today, the North Star programme represents the world’s largest natural history study (a study that looks how the condition changes over time) of over 2,000 boys and men living with DMD, as well as a clinical network of healthcare professionals across 23 centres in the UK, that focuses on the best clinical management.
The NorthStar network currently includes over 250 participating members, including 74 clinicians, 11 registrars/clinical fellows, 79 physiotherapists and 52 nurses or occupational therapists.
Celebrating significant milestones
During the past two decades, the programme has reached significant milestones, which led to improvements not only of the design of clinical trials, but also of the management of DMD and the quality of life of people living with the condition.
Development of the NorthStar Ambulatory Assessment (NSAA) in 2011 resulted in creation of 17 movement tests to help monitor the progression of the condition. These tests evaluate the movement abilities of boys who can walk. Along with monitoring the progression of DMD, it is also widely used in assessing the effectiveness of treatments in clinical trials.
In 2013, the clinicians from the NorthStar Clinical Network reported on the long-term benefits, and associated side effects, of using steroids to manage DMD symptoms.
NorthStar Programme celebrates milestones achieved over 20 years – Credit: Muscular Dystrophy UK
Another important milestone achieved by NorthStar has been setting up the adult NorthStar Network, led by Professor Ros Quinlivan from University College London Hospital. This has led to the development of the first-ever clinical care guidelines for adults with DMD.
Improving standards
“ “The NorthStar Network was established 20 years ago to help improve standards of care for boys with Duchenne muscular dystrophy across the entire country. Today, the improved standards have helped many boys living with the condition extend their lives into adulthood.
“Muscular Dystrophy UK’s support has helped us to establish standardised outcome measures that have now been adopted globally both in the regular clinics and to assess efficacy of therapies, driving faster more effective research. We’re proud of how far we have come and excited for the future.” ”
Professor Francesco Muntoni
Paediatric Neurologist at Great Ormond Street Hospital, and Chief Investigator for NorthStar
“We’re proud to have been supporting the NorthStar programme over the past 20 years. The work has played an instrumental role in improving the quality of life for boys and men living with the condition and influencing best practice in care nationally.
“It’s also helping to accelerate the development of treatments, by providing data to improve the design and evaluation of clinical trials. With the hope to overall improve quality of life, with a promise of a better future.”
Dr Kate Adcock
Our director of research and innovation
Helping future generations
Darren Driffield with son Joe celebrates 20 years of NorthStar. Credit: Chris O'Donovan/Muscular Dystrophy UK
“When Joe was on a clinical trial led by the team at the John Walton Muscular Dystrophy Research Centre, the NorthStar Ambulatory Assessment helped us, as parents, understand the benefits of the treatment he was receiving. Further investment in the NorthStar Programme has led to better standards of care for boys, like Joe, and more recently adults living with Duchenne. Their research into the progression of the condition could ultimately lead to new treatments becoming available in the future.”
Darren Driffield
Father, his 14-year-old son, Joe, was diagnosed with DMD in 2014
A shining light
The NorthStar programme got its name after Michael McGrath, who lives with a muscle wasting condition himself, reached the North Pole in his endeavours to raise money for muscular dystrophies.
“I recall speaking with the distinguished Lord John Walton of Detchant and Professor Francesco Muntoni at the launch of the North Star programme. Whilst our conversation touched on the standardisation of care and the importance of data capture, which NorthStar has I believe achieved, I remember vividly talking about the project’s name which describes the closest star to earth’s North celestial pole. I hope I was able to play a small part in its establishment and success. After all these years Muscular Dystrophy UK is a charity that still to this day remains close to my heart.”
Michael McGrath
Founder and CEO of Muscle Help Foundation
Northstar Programme
The NorthStar programme is made up of two parts. It’s the world’s largest natural history study of boys and men with Duchenne muscular dystrophy (DMD) and a clinical network of UK-wide healthcare professionals focusing on the best clinical management of DMD.
We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster. Help us fund groundbreaking research and life-changing support. Together we can change the future of muscle wasting conditions.
