1 Report of the Board of Trustees

The Board of Trustees of Muscular Dystrophy UK (the operating name of the Muscular Dystrophy Group of Great Britain and Northern Ireland) presents its Annual Report and Financial Statements for the year ended 31 March 2020.

What Muscular Dystrophy UK does – core purpose and activities

Our vision A world with effective treatments and cures for all muscle-wasting conditions and no limits in life for individuals and families affected.

Our mission We are the UK charity for individuals and families living with muscle-wasting conditions. • We support research to drive the development of effective treatments and cures. • We ensure access to specialist NHS care and support. • We provide services and promote opportunities to enable individuals and their families to live as independently as possible.

We know we can beat muscle-wasting conditions more quickly if we work together. We are uniting skills, knowledge and resources in the UK and working with others around the world so we can improve the quality of life for those affected, and bring treatments and cures closer to reality.

Our values • We care: we care about everyone affected by muscle-wasting conditions. • We are inclusive: we are working to ensure there is support and treatment for everyone living with muscle-wasting conditions in the UK. • We are collaborative: we work in partnership across the UK, and internationally, to maximise our impact. • We are focused on results: everything we do is designed to improve the quality of life for individuals and families; we won’t stop until treatments and cures are found for all muscle-wasting conditions. • We are determined: we have been leading the fight against muscle-wasting conditions for more than 60 years and are unwavering in our commitment to unite resources and achieve our vision.

Objects of Muscular Dystrophy UK for the Public Benefit The charity is established to promote research into muscle-wasting conditions; to promote the provision of care and treatments to those affected; to assist those who care for individuals affected by such conditions; to promote education and training for affected individuals and to raise the awareness of the public on any matter relating to the charity’s objects.

Public Benefit The charity Trustees consider that they have complied with their duty in section 17 of the Charities Act 2011 to have due regard to Public Benefit guidance published by the Charity Commission and that the benefits that the charity provides are not unreasonably restricted. The activities and impact described in the report below all contribute to the public benefit delivered by the charity.

The work of Muscular Dystrophy UK Muscle-wasting conditions are a group of rare and very rare conditions. Compared with the more common, acute conditions, they do not receive the same priority in research funding or investment in clinical care.

Muscular Dystrophy UK (MDUK) works to improve the lives of an estimated 70,000 people living with a muscle-wasting or associated condition today. We believe this as passionately now as we did when we were established over 60 years ago. It drives everything we do.

It is this vision that unites us all – from researchers we fund to the health professionals that we support from the people we help through our information and advocacy services to our supporters who inform, guide and campaign with us to make change happen.

We: • fund pioneering research for better treatments that will improve people’s lives today and transform those of future generations drive change so that people have faster access to treatments, as well as the best NHS care and support ensure that we are here for all people and their families affected by a muscle-wasting condition whether awaiting a diagnosis, recently diagnosed or living with a condition.

Over the years, the charity has made important contributions in funding research and developing clinical support leading to vital progress such as an early and accurate diagnosis, access to steroid treatments, spinal surgery and respiratory support, which, with improved care, enhances quality of life and life expectancy.

With advances in technology, there are now opportunities to achieve a great deal more through identifying and developing promising research, helping to get clinical trials underway and developing a critical mass of scientists and clinicians working together to tackle the challenge of muscle-wasting conditions.

Achievements and performance against objectives for 2019/20

Overview

Performance Indicators

To support research with the goal of effective treatments and cures being identified for all conditions

To ensure access to specialist NHS care from a multi-disciplinary team

To provide services and promote opportunities to enable each affected individual to live as independent a life as they wish

Income generation Progress during the year

In 2019/20 we continued our support for highquality research and committed £1.2m into nine new research projects as well as £1.3m to support UK clinical trial capacity and other activities that underpin research into neuromuscular conditions. We continue to provide information on current research developments and clinical trials through our rebranded Research Line, bringing the total research commitment to over £10m.

We successfully secured NHS funding for 11 neuromuscular specialist posts, supported clinical networks, and provided training to over 2,600 through conferences and online training modules to better equip them to meet the needs of people affected by muscle-wasting conditions.

We responded to over 3,200 requests for information or support over the phone, email and face-to-face, and 22,000 info factsheets were downloaded from our website. We saw a seven percent increase on the previous year in the number of people we supported through our advocacy service, securing over £1.5m in services for them.

We were on track to achieve the total budgeted income target for this year but owing to the impact of the COVID-19 pandemic on our work, we raised £6.2m. This was £383k below our anticipated fundraising budget. We developed a new regional strategy to focus on the audiences that have the greatest reason to support MDUK, in order to develop long-term and meaningful relationships with them. This growing of grassroots support, focussing on localised fundraising groups and Family Funds, meant we were in a good position when the impact of COVID-19 hit.

Improving lives today and transforming those of the future: accelerating research to develop effective treatments and cures

MDUK works to improve the lives of people living with a muscle-wasting or associated condition. High-quality research plays a key role in our ambition, helping us to better understand these conditions and maximise treatment improvements.

Our three-year research strategy ‘Transforming lives through research’ provides the guiding force for our research support. We have continued to invest in high-quality research to deepen our understanding of musclewasting conditions and support studies into ultra-rare conditions. We have begun the journey to encourage new researchers to join the field. In addition, we have continued to work to expand patient access to clinical trials.

We entered into a new funding relationship with SMA UK to jointly support PhD students in our Grant Round 2020. Unfortunately, we had to cancel the Grant Round 2020 as a result of the COVID-19 pandemic. Nevertheless, we continue to maintain relationships with funders such as the Medical Research Council, the National Institute of Health Research and others, to develop new opportunities for joint working.

MDUK is an executive member of the European Neuromuscular Centre (ENMC). This organisation was set up over 25 years ago to “encourage and facilitate communication and collaboration in the field of neuromuscular research with the aim of improving diagnosis and prognosis, finding effective treatments and optimising standards of care to improve the quality of life of people affected by neuromuscular disorders”. Members of the ENMC are national, neuromuscular research charities from across Europe. The ENMC supported eight workshops in 2019/20, seven of which included UK clinicians, researchers and patient representatives. This could not have happened without MDUK’s support.

In 2019/20, we reviewed and streamlined our grant-making and grant scheme processes. In consultation with our Medical Research Committee, we have amended the eligibility for applicants. As of the Grant Round 2020, each applicant can submit up to one grant application per grant type; multiple grant applications for the same grant type will not be accepted. In addition, a single resubmission of a previously turneddown MDUK application is permitted.

We awarded nine new research projects and PhD studentships in 2019/20. These grants address aspects that fit with the four priority areas outlined in our research strategy, namely: harnessing the power of genetics; understanding disease mechanisms; facilitating treatment development; and improving quality of life and wellbeing. Some of these projects will investigate methods to improve the delivery of drugs, optimise the use of cough-assist devices and

find out how many people are living with muscle-wasting conditions in the UK. The charity is now funding around 50 different projects in the UK and through international partnerships.

Through our individualised service, the MDUK Research Line, we have continued to ensure that patients and families can find out about new studies and treatments and clinical trials.