Our projects

Brilliant scientists, powerful collaborations. We find and fund projects that are ripe for treating Duchenne now.

Newly funded projects

LTBP4

Modifying the protein LTBP4 to target inflammation and fibrosis – two key challenges in Duchenne.

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Non-invasive imaging biomarker

Reducing biopsies and improving dystrophin analysis in Duchenne boys.

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Blood-based biomarker

Measuring disease progression and response to new treatments.

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Pre-clinical research

Solid GT

A ground-breaking programme for exploring gene therapy for Duchenne.

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Vamorolone

An alternative to existing steroid treatments for Duchenne.

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Clinical research and support posts

Dr Michela Guglieri

Newcastle Clinical Research Fellow

Cathy Turner

Treat-NMD Duchenne Programme Co-ordinator

Awards for up-and-coming researchers

Kathy Wang

HHMI Medical Research Fellowship

Dr Michel Abou Samra

World Muscle Society Congress prize

Some of our past projects

Ringo

Dystrophic dogs provide insights into Duchenne research.

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HT-100

Pre-clinical research for drug to target muscle fibrosis.

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SMT C1100

Increasing utrophin to compensate for dystrophin.

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Our mission

We aim to stop Duchenne muscular dystrophy being a death sentence for hundreds of thousands of boys worldwide.

About us

Our impact

We are helping turn the hope of treatments into reality. Critical supportive drugs are in clinical trial thanks to our funds.

Latest news

Your help

In the last 3 years we have given more than £1.5m to ground-breaking research projects.
And there is still work to do.

Donate now