Our projects

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

Current projects

Neurobehavioural issues in Duchenne

Tackling learning, behaviour, depression and anxiety.

DMD Care UK

Access to best care for everyone with Duchenne.

Combating immunity to AAV

Ensuring as many patients as possible can benefit from gene therapy.

Solid GT

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

Vamorolone

An alternative to existing steroid treatments for Duchenne.

CRISPR for Duchenne

Gene editing technology to correct mutated dystrophin gene.

Gene therapy for Becker

A microdystrophin gene therapy approach for Becker muscular dystrophy.

Activity monitoring biomarker

KINEDMD: Wearable sensor technology to monitor Duchenne patients’ activity.

Non-viral delivery

A next-generation gene therapy delivery approach.

Grants to support clinical and research staff and facilities

Dr Michela Guglieri

Newcastle Senior Clinical Lecturer and Neurologist

Manju Agarwal

Clinical trial co-ordinator at Great Ormond Street Hospital

Nicola Burnett

Senior physiotherapist, Great Ormond Street Hospital

Cathy Turner

Treat-NMD Duchenne Programme Co-ordinator

Refurbishment and expansion of paediatric space

Clinical Research Facility at Newcastle Royal Infirmary

Awards for up-and-coming researchers

Kathy Wang

HHMI Medical Research Fellowship

Eleonora Maino

World Muscle Society Congress prize

Research projects

Blood-based biomarker

Measuring disease progression and response to new treatments.

Ringo

Dystrophic dogs provide insights into Duchenne research.

LTBP4

This project focuses on targeting inflammation and fibrosis – two key challenges in Duchenne.

HT-100

Pre-clinical research for drug to target muscle fibrosis.

Non-invasive imaging biomarker

Reducing biopsies and improving dystrophin analysis in Duchenne boys.

SMT C1100

Increasing utrophin to compensate for dystrophin.

Our commitment

We are passionate about supporting the most promising research to find therapies and a cure for Duchenne muscular dystrophy. We aim to focus our efforts on tackling Duchenne for all sufferers, regardless of their type of mutation. We have an open grant call and all applications are peer reviewed.

Our scientific advisory board comprises leading minds in the field: Professor Ronald Cohn, Dr Kevin Campbell, Professor Madeleine Durbeej-Hjalt and Dr James Dowling together with our Honorary Scientific Patron Professor Victor Dubowitz. Read more about their work here. Please contact research [at] duchenne.org.uk for more information about applications.

Our mission

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

Our impact

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

Your help

Your generosity has funded millions of pounds worth of ground-breaking research.
And there is still work to do.