NorthStar: 20 Years of Support for People with Duchenne Muscular Dystrophy

MUSCULAR Dystrophy UK marks 20 years of the NorthStar programme, designed to gain a better understanding of Duchenne muscular dystrophy (DMD).

Muscular Dystrophy UK is the leading charity for more than 110,000 children and adults in the UK living with one of over 60 muscle wasting and weakening conditions. It has invested more than £1.5 million in the NorthStar programme since it first began in 2004.

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.

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.  About 100 boys with DMD are born in the UK each year and there are about 2,500 boys and men known to be living with the condition in the UK at any one time.

Today, the North Star programme represents the world’s largest natural history study (a study that looks at 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.

During the past two decades, the programme has reached significant milestones, which led to improvements not only in the design of clinical trials but also in the management of DMD and the quality of life of people living with the condition.

The development of the NorthStar Ambulatory Assessment (NSAA) in 2011 has seen the implementation 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.

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.

Professor Muntoni said:

“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 the efficacy of therapies, driving faster more effective research. We’re proud of how far we have come and excited for the future.”

Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK said:

“Muscular Dystrophy UK is 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 practices 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 improve the quality of life overall, and with a promise of a better future.”

Darren Driffield, whose 14-year-old son, Joe, was diagnosed with Duchenne muscular dystrophy in 2014 said:

“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.”

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 dystrophy.

For further information about the NorthStar Programme, please visit: musculardystrophyuk.org/NorthStar.