Inspiring Business by Sharing Success

Fill Our Heart with Hope: Alex’s Wish Launches New Campaign

Fill Our Heart with Hope: Alex’s Wish Launches New Campaign

Leicestershire based charity, Alex’s Wish has announced that it will be working with Duchenne UK to raise money for a grant call out to the world’s best scientists and researchers to understand how the heart is affected by DMD (Duchenne Muscular Dystrophy) and identify potential solutions.

To do this, the charity needs to raise £80,000. Its Autumn Charity Ball raised £33,000, so the 'Fill our Heart with Hope' Campaign has been launched to raise the remaining £47,000.

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.

The 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, launching this month (March 2024) for six weeks, to expression of interest applications. More details on the scope and eligibility will be published in due course.

The Science

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.

< Back