This Trustee’s Week (6 - 10 November 2023) we’re delighted to announce three new Trustee appointments.

We welcome Lord Sharkey, James Lee and Gerry McMenemy, who will join our existing Trustees to set our strategic direction, monitor the delivery of our objectives, uphold our values to governance and guide, advise and support us in achieving our overall vision and purpose.

Meet our Trustees

Lord Sharkey

John Sharkey has spent most of his commercial life in communications businesses. He was joint managing director of Saatchi and Saatchi UK, founded and ran his own advertising agency and was chair of various other communications and recruitment enterprises. He became strategic communications advisor to Nick Clegg in 2007 and was chair of the Liberal Democrat’s General Election campaign in 2010 and of the 2011 Fairer Votes campaign.

A former chair of the Association of Medical Research Charities, which he chaired for seven years, he is now a member of the House of Lords and of its Science and Technology Committee.

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Gerry McMenemy

Gerry became involved with the charity shortly after his son was diagnosed with Duchenne muscular dystrophy. He and his wife appreciated the support of the charity, so they joined as a member, launched the family fund ‘Muscle in with Somhairle’ and Gerry became a member of the charity’s Scottish Council. He brings his lived experience as a parent and ambassador of a child with a muscle wasting condition, and his skills as an accomplished communicator and negotiator from his role as a Scottish Police Officer working through a variety of front line, operational, critical, and specialist policing fields in both management and leadership roles.

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James Lee

James was diagnosed with Spinal muscular atrophy (SMA type 3) as a small child and has both volunteered and worked at Muscular Dystrophy UK. He is currently Head of the Bridge Programme at the City Bridge Trust and a former Chair of the Grants Committee at BBC Children in Need. He also sits on the Disability Advisory Group working with Transport for London and is a member of the London Recovery Board for the Greater London Authority.

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A warm welcome

Chair of Trustees, Professor Michael Hanna said:

It’s a pleasure to welcome our new trustees, who join us with a wealth and breadth of experience, knowledge and expertise.

Our Chief Executive, Catherine Woodhead, said:

The support that Muscular Dystrophy UK receives from our dedicated Trustees is paramount in helping us to make great advances, which would have been unthinkable just 10 years ago.

Trustee’s Week is an opportunity to give a warm welcome to Lord Sharkey, James Lee and Gerry McMenemy and thank our existing Trustees for playing a vital part in supporting the muscle wasting and weakening community. Together we are stronger.

A special thanks to Marcus Brown, Tanvi Vyas and Michael Armstrong, who’ve recently stepped down from their role as Trustee. We wish them all the very best.

Interested in becoming a Trustee for us? Find out how you can apply and help make a difference to the muscle wasting and weaking community.

UPDATE (3 November 2023):

A webinar regarding the EMBARK study results for families took place online via Zoom on 2 November. It was organised by the Northstar Network and support by us, Action Duchenne and Duchenne UK. 

(31 October):

On Monday 30 October 2023, Sarepta Therapeutics announced the one-year results of part 1 of the EMBARK Phase 3 clinical trial. This multinational, randomised, controlled, and double-blind study aims to assess the safety and efficacy of delandistrogene moxeparvovec, a gene therapy now known as Elevidys (previously called SRP-9001) in boys aged four-seven with Duchenne muscular dystrophy (DMD).

The results show that the study failed to achieve its primary objective (also known as endpoint), which was to demonstrate a significant difference in the change of the North Star Ambulatory Assessment (NSAA) total score following Elevidys treatment. The NSAA is a recognised way of evaluating motor function (the ability to move) in people living with DMD. Although some improvement in motor function was observed in children treated with Elevidys compared to the placebo group, these differences were not statistically significant.

The study did successfully meet two of its secondary endpoints -the time required to rise from the floor and the 10-metre walk test. These secondary measures showed significant improvements in children who received Elevidys over the course of one year.

Additionally, there were no new safety concerns raised during this part of the study.

The study involved 125 boys with DMD who can walk unassisted, and who were randomly assigned to either the treatment group or the placebo group. Participants will continue to be closely monitored for the next five years for both safety and clinical outcomes.

Part 2 of the EMBARK Phase 3 study is currently ongoing and should finish late 2024, when it will provide further insights into the safety and efficacy of Elevidys in boys with DMD.

There will be a webinar for families in the coming days with members of the UK paediatric NorthStar network and other patient organisations – Action Duchenne and Duchenne UK. We will share more about the webinar when the details are known. We also remain in communication with Roche, Sarepta's global distribution partner, and will provide ongoing updates. In the meantime, you can read a community letter from Roche about the study and what it means for people in the UK.

Notably, Elevidys received what is known as accelerate approval for boys aged four-five in the United States earlier this year. The health authority in the UK responsible for issuing marketing authorisations (the licence) for medicines is the Medicines & Healthcare Products Regulatory Agency (MHRA), not the FDA. Roche is committed to working with the relevant agencies to determine the best possible route to achieving a potential licence for Elevidys.

We’re thrilled to announce that we’ll be at the RHS Chelsea Flower Show next year! Our Show Garden on Main Avenue is fully funded by grant-making charity Project Giving Back and designed by the RHS ’Young Designer of the Year’ 2017, Ula Maria.

The 'Muscular Dystrophy UK - Forest Bathing Garden' will offer an accessible, immersive forest bathing experience featuring more than 50 birch trees. 4,000 plants, the majority of which have been selected for their beautiful foliage, will create a green tapestry that is rich in texture, with an occasional burst of colour.

Forest bathing, otherwise known as Shinrin-yoku, is an ancient Japanese practice of spending time in the forest and soaking up its atmosphere through the senses. The garden seeks to create a sheltered space for its visitors – to give comfort and clarity, reconnect with oneself and nature, or accommodate conversation with others.

Visitors will access the garden through an accessible path that follows a slow-moving naturalised water stream. At the heart of the garden is a central meeting hub with informal seating and sculptural knapped flint walls that will provide a sheltered space for people to meet. The random knapped flint pattern was chosen by Ula due to its beautiful texture and form that is reminiscent of muscle cells.

A key feature of the garden is a large bungaroosh-style wall, which will be made using modular steelwork sections filled with a mix of reclaimed and recycled materials such as large stone blocks, slate tiles, and bricks. This building technique was chosen to showcase how a beautiful and contemporary-looking garden structure can be created using a variety of reclaimed materials.

After being displayed at the RHS Chelsea Flower Show from 20-25 May 2024, the garden will be relocated to the public garden space at the new Institute of Developmental & Regenerative Medicine at the University of Oxford. There it will be a permanent place of refuge for patients during treatment and new trials, as well as clinicians, scientists and researchers and with access to the general public too. 

Oxford holds a special significance for us, as the charity has been supporting scientific research in the city for over five decades and we hold our annual Bidwells Town and Gown 10k, which draws 5,000 runners, in the city.

Designer Ula Maria said: 

This garden is all about connections: whether to oneself, nature, or others. It is meant to serve as a sanctuary whilst offering an immersive forest bathing experience. It is my hope that this garden will increase awareness of how places that are inspired by the people who inhabit them can have a meaningful effect on communities such as Muscular Dystrophy UK.

Catherine Woodhead, Chief Executive of Muscular Dystrophy UK said:

Thanks to the generous sponsorship from Project Giving Back, we are absolutely delighted to have a garden at the RHS Chelsea Flower Show 2024. We want more people to know about Muscular Dystrophy and what it’s like to live with a muscle-wasting or weakening condition. Part of the challenge of a diagnosis is that no one has heard of it, you know no-one living with it and you have to explain it to everyone from family, friends, teachers, colleagues and even GPs. This opportunity will change that. It will allow us to reach people who have never heard of the condition, give our community a voice and raise awareness of the work of the charity.

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Calling all researchers – we’re pleased to announce the 2024 Grant Round for research proposals opens today (Monday 9 October).

Researchers can apply for funding to support PhD studentships and project grants that have relevance to one or more type of muscle wasting condition. Specific funds are available for projects related to facioscapulohumeral muscular dystrophy (FSHD), limb girdle muscular dystrophy (LGMD) and LMNA congenital muscular dystrophy and we would especially welcome applications in these areas. However, we will fund research across the breadth of conditions supported by the charity, including rare and very-rare conditions.

The deadline for submission of the online application is 3pm Wednesday 17 January 2024.

Following a period of rigorous review, funding decisions will be made in July 2024, and we will announce the research projects we are funding in Autumn 2024.

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

Just last month we announced we had awarded 11 new grants worth over £1.3m as part of our 2023 grant round so it’s great that we are now looking forward to 2024.

We want to invest in research that increases our understanding of neuromuscular conditions and that promotes the development of potential treatments. We also welcome applications that will have a direct, positive impact on the quality of life of people with muscle wasting or weakening conditions or will help establish best practice in symptom management