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Campaign

Autumn 2024

The newsletter for our supporters.

Welcome

I’m delighted to welcome you to this year’s autumn edition, showing how your support helps us be there for people living with muscle wasting conditions. I was pleased to visit our award-winning Forest Bathing Garden at the RHS Chelsea Flower Show in May, where we spoke to many visitors, many of whom hadn’t heard of muscular dystrophy or our work.

We recently carried out a survey to better understand the priorities of our community, with one key finding being the need to raise awareness of muscle wasting conditions among health professionals, which will be a focus for our work going forward.

You can also read about Sarah’s decade-long journey to a diagnosis of limb girdle muscular dystrophy, an example of the challenges we aim to address.

Thank you for your support – it makes our work possible.

Gabby Logan MBE
President, Muscular Dystrophy UK

A happy women and boy crouch by and interact with a water feature at Chelsea Flower Show in MDULK's garden.

Success at Chelsea Flower Show

We were thrilled to have a garden at this year’s RHS Chelsea Flower Show on Main Avenue, designed by Ula Maria, 2017 RHS Young Designer of the Year, and fully funded by Project Giving Back. We were incredibly proud to not only win a gold medal but also be awarded Best Show Garden, which led to unprecedented media coverage and a significant increase in website visits.

The Muscular Dystrophy UK Forest Bathing Garden was designed as a peaceful, accessible space with people living with muscle wasting conditions in mind.

After the show, the garden will be relocated to the Prince and Princess of Wales Hospice in Glasgow, where it will offer a unique outdoor space for patients, families, and the local community.

We also used this opportunity to engage with health professionals and raise awareness.

Research News

We’re making great progress. But there is still so much that needs to be done. Donate today.

An important step forward to improve our understanding of nemaline myopathies

We’ve awarded almost £600,000 to Professor Laurent Servais at the University of Oxford to carry out a natural history study of people living with nemaline myopathies.

He will be tracking the progression of nemaline myopathies across multiple UK sites, with a goal of recruiting 45 patients to help shape future research and clinical trials.

Investigating molecular patch approaches for a collagen VI-related muscular dystrophy.

We’re awarded £75,000 to Professor Carsten Bonnemann and his team to develop a new exon-skipping therapy for collagen VI-related muscular dystrophy, using adeno-associated viruses to deliver genetic material into cells.

The “pseudoexon-skipping” approach aims to target a genetic change in COL6A1, which causes muscle damage and severe forms of collagen VI-related muscular dystrophy, with the potential to improve outcomes for those living with Bethlem myopathy and Ullrich congenital muscular dystrophy.

Testing new drugs for spinal muscular atrophy

While recent treatments for Spinal muscular atrophy (SMA) have emerged, they are not complete cures.

We’re funding a study to explore drug repositioning, identifying existing drugs approved for other diseases that could help treat SMA by profiling molecular effects, with the aim of improving future treatments and easing the management of the condition.

This piece is written by Bill Duddy, a senior lecturer in the neuromuscular research team at Ulster University School of Medicine, who, after losing his brother to Duchenne muscular dystrophy, has dedicated his career to understanding and treating neuromuscular conditions. Read Bills story.

A women wearing a pretty summer scarf sits in the sun

A helping hand

To support those living with muscle-wasting conditions, we train and develop Peer Support Volunteers. Joan shares how Muscular Dystrophy UK has been there for her over the years and how rewarding she finds helping others as a Peer Support Volunteer.

Through her experience, Joan has connected with others facing similar challenges, offering advice and emotional support, whether it’s about working with a muscle wasting condition or discussing treatment options like Risdiplam.

Your generous support allows us to provide vital services like the Peer Support Service, with full training offered to all our volunteers.

Understanding our community‘s priorities better

At the end of 2023, we launched a community survey to better understand the priorities of those living with muscle-wasting conditions and those supporting them. A key finding being the need to raise awareness among healthcare professionals:

Delays in diagnosis

It took four or more meetings with health professionals for the majority, 55%, of respondents to get a diagnosis.

Misdiagnosis

Just under half, 47%, faced being misdiagnosed at some point.

Long waits

A significant majority, 69%, waited more than a year to receive a diagnosis.

Key priority

84% said increasing the understanding and awareness of muscle wasting and weakening conditions was a key priority.

A photo of a lady sitting in front of a green leafy background

Sarah’s Story

Sarah’s journey to a diagnosis of limb girdle muscular dystrophy (LGMD) type 2A took over a decade. Despite early struggles with sports and numerous doctor visits, her condition remained undiagnosed, with doctors offering various explanations.

After years of pushing through, Sarah decided to revisit her biopsy from 1999, and with advances in science, it was finally confirmed that she had LGMD type 2A. This long wait led to depression, but with counselling and support, she rebuilt her life and now works in the NHS, where her needs are supported.

Your kind support enables us to engage with health professionals to ensure fewer people experience the delays that Sarah faced.

A man and women talk in a sea of other people.

Acting on our communities priorities

As highlighted in our community survey, over 80% of respondents emphasized the importance of increasing understanding of muscle-wasting conditions among non-neuromuscular specialist health professionals.

To address this, we held a successful engagement event at the RHS Chelsea Flower Show, connecting with over 100 health professionals and promoting training opportunities to enhance their knowledge.

This event, along with our Regional Neuromuscular networks and upcoming webinars, plays a vital role in improving support for those living with these conditions.

Your generous donations help us run such events, enabling us to continue raising awareness and offering essential resources.

Campaigning for access to treament

For people with muscle-wasting conditions, every day counts, and with many clinical trials and treatments on the horizon, our key campaigning priority is ensuring access to these new treatments.

Your support enables us to lead advocacy efforts, gather powerful community stories, and ensure the voices of people living with muscle wasting and weakening conditions are heard. But there is still work to be done.

We are funding clinical trials for a variety of muscle wasting conditions. Additionally, we’re working to ensure access to seven treatments for three different conditions that have already completed clinical trials.

Please help us continue this vital work and make sure no one is left behind.

Get involved

Christmas Shop

Be one of the first to check out our festive selection of Christmas cards, gifts, scented candles and so much more.

Christmas Raffle

Our Christmas Raffle is a great way to support our life-changing work AND to be in with a chance to win a fantastic cash prize, just in time for Christmas.

What would you do if you won our £2,500 top prize?

Give as you live

If you shop online, you can raise free funds for us while you shop! Simply join Give As You Live (GAYL) to start raising free donations for us, at no cost to yourself.

Lottery

Help change the future for muscle wasting conditions for children like Carmela (above) – as well as have a chance to win fantastic cash prizes – by entering our Weekly Lottery!

Campaign Magazine

Twice a year Campaign magazine brings you inspiring stories, research breakthroughs, and updates on how your support is making a difference.

Join Friends of MDUK today to receive your printed copy of Campaign!