Our research strategy

Every day we’re working towards a future with effective treatments and ultimately cures for all muscle wasting and associated neuromuscular conditions. We believe this as passionately now as we did when we were established in 1959. 

It is this vision that unites us all and continues to drive our research strategy. 

We fund pioneering research for better treatments to improve people’s lives today, and to transform those of future generations. Every day scientists are gaining new knowledge of muscle wasting conditions, so there are always exciting new treatments on the horizon. 

The theme of innovation and partnership underpins all that we do and enables research to be given every chance to succeed. By supporting research in the UK and across the world, we link researchers and research projects, accelerating scientific progress and helping us to improve lives as quickly as possible. 

Many conditions are regarded as very rare, but research is working towards new treatment possibilities all the time. For example, scientists are building on what they have learnt from developing therapies for conditions such as Duchenne muscular dystrophy (DMD) and spinal muscular atrophy. Some of these findings are now taking us towards more targeted treatments for DMD, collagen-VI muscular dystrophy and mitochondrial diseases. 

Deepen our understanding of neuromuscular conditions 

We need to deepen our understanding of neuromuscular conditions in order to develop potential treatments and ensure that there is a pipeline for clinical trials. We will continue to support excellent scientists in carrying out first class, fundamental research into neuromuscular conditions. 

Support more studies into very rare conditions 

We will work with international funders and charities to ensure that we bring together research skills from across the globe. By collaborating internationally, researchers can reach larger numbers of patients, which will speed up the journey to develop new treatments. 

Facilitate clinical trial readiness 

There is a growth in potential treatments becoming available for neuromuscular conditions. It is, therefore, vital that researchers have enough information about the symptoms and biology (natural history) to design clinical trials and show the regulators that a treatment is beneficial. To help facilitate clinical trial readiness, will support studies that help us understand the natural history of conditions and provide insight into the best outcomes to measure to show treatment effectiveness.  

Encourage new researchers to join the field 

To support neuromuscular research in the long term, we need to expand the existing pool of dedicated and talented researchers. We will support and encourage researchers early on in their careers, as well as experienced researchers from other fields, to join the neuromuscular community. 

Supporting allied healthcare professionals 

Allied healthcare professionals including physiotherapists, orthotists, occupational therapists, speech and language therapists and specialist nursing staff are all key to understanding the impact of a muscle wasting condition and improving quality of life for those affected. We will provide further opportunities for quality-of-life research through our grant schemes. 

The theme of innovation and partnership underpins all that we do and enables research to be given every chance to succeed. 

Harnessing the power of genetics. So that:

• we improve our understanding why people with the same condition are affected differently. 
• more people receive a confirmed genetic diagnosis, which will help us better understand the conditions. 

Understanding disease mechanisms. By: 

• developing new models that mimic the human condition that can be used to assess the potential of new drugs. 
• investigating what happens inside the body, not only within muscles but also in other organs such as the brain or the gut. 

Facilitating treatment development. We will: 

• support patient registries and studies that assess how a condition progresses with time (natural history studies), as these are valuable resources for drug companies and other researchers. 
• work across the research community to encourage and support collaboration to speed up the journey to develop new treatments. 

Improving quality of life. We will: 

• develop evidence-based interventions that improve the quality of life of people affected by muscle wasting conditions, putting the patients’ knowledge and experiences at the heart of the research agenda. 
• better understand the psychological impact of living with muscle wasting conditions. 
• investigate the management of fatigue, pain and other aspects that impact on the quality of life experienced by people living with muscle wasting conditions. 

 Investing in the future we will: 

• continue to invest money in high-quality research to understand the underlying causes of muscle wasting conditions, and to develop potential treatments, always making sure our investment has maximum impact. Through our annual grant round we currently support many grants that help us deliver our research strategy. However, there are aspects that can be accelerated if we make some strategically placed investments. 
• encourage scientists with new ideas and new technologies to come into this area of research, building on existing scientific study to develop a new generation of therapies, with the ultimate goal of being able to treat all people, whatever their muscle wasting condition 
• use seed funding to drive innovative research to enhance the quality of life for people living with muscle wasting conditions.