Fill our Hearts with Hope

February 15, 2024 4:08 pm

February is the month of LOVE, so what a perfect time to launch our HEART campaign…..

Our heart muscles need dystrophin to function efficiently. In DMD, a lack of dystrophin means that heart muscles weaken over time. Heart cells are replaced by scar and fatty tissue, which leads to a type of heart muscle disease called dilated cardiomyopathy

The age when cardiomyopathy happens in DMD can vary from person to person, from early childhood to adulthood.  As Professor John Bourke, Consultant Cardiologist said “the heart is now the critical determinant of survival in DMD”

Children with DMD don’t usually have cardiac symptoms.  However, weakening heart muscles are part of the natural progression of the disease.  Therefore, early monitoring, preventative treatment, and ongoing management are important for keeping the heart healthy into adulthood.  It is currently not possible to prevent heart weakness in DMD completely.  However, Steroids and Heart Medication can slow down the decline in heart function.

This year we are working with Duchenne UK to raise money for a grant call out to the worlds best scientists and researchers to start working on this problem, understand how the heart is affected and identify potential solutions.

To do this we need to raise £80,000, our Autumn Charity Ball raised £33,000 but we would like to raise another £47,000 – so we have launched our ‘Fill our Heart with Hope’ Campaign.

What Duchenne UK is looking to fund
Duchenne UK will fund ambitious, translational or clinical projects which address the following areas

  • Understanding the unique features of the DMD heart and cardiac disease progression in DMD
  • Innovative treatment strategies and approaches (e.g. who and when to treat, young patient, early disease stage versus older patients, advanced disease stage), in the context of the latest clinical recommendations and cardiac standard of care for DMD
  • How to evaluate the cardiac response to therapies better
  • Characterisation of heart phenotypes in DMD to help understand the efficacy and safety profile of disease modifying treatments

Two funding strands
To maximise the impact of its funding, Duchenne UK has divided the call for research projects into two strands:

  • Strand one – ‘Help the Heart’: This cardiac themed open research call will run until the end of 2024 with a broad scope, but focused on the cardiac unmet needs in DMD, using Duchenne UK response-mode funding model. Please find more details here.
  • Strand two – A therapeutic focused call: This will launch in March for six weeks, to expression of interest applications. More details on the scope and eligibility will be published in due course.

Help the heart
Inviting applications, Emily Reuben OBE, Co-founder and Chief Executive of Duchenne UK, says:

“We are delighted to be launching this ambitious grant call, which we hope will draw out new treatments for cardiac care. This is a critical area for people with DMD, and we hope to be able to encourage the development of new treatments to help the heart. We are hugely grateful to our partner funders to enable us to offer a transformative amount of funding for this project”

Emma Hallam, Founder of Alex’s Wish, and mum to Alex aged 17

“With a teenager living with Duchenne ourselves, we understand first-hand the priority of heart health.  Time and time again, we hear of boys losing their lives far too soon of heart failure.  This has to stop, we are incredibly proud of supporting this important work to help save and extend lives of everyone affected by Duchenne.”

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