Our Impact

We exist to

Defeat

Duchenne

for this generation of boys

Our approach is simple: fund the best science, support practical clinical trials, and make sure boys in the UK can access treatment and care, wherever they live.

2013-2026

at a glance

£2.9m

raised to date

22

medical research projects funded

6

clinical trial projects funded

5

projects funded – improving clinical trial outcomes

4

projects funded – improving clinical trial capacity

2

projects funded –
new technologies

4

projects funded – improving care standards

1

Research PHD post funded and filled

5,000+

smiles raised to date (give or take a few)

Funding

What We Fund

We invest across the full treatment journey –
from research to real-world care.

Medical Research & Trials
  • Gene therapy and gene editing
  • Stem cell therapy
  • Heart research and early detection
  • Steroid optimisation and alternatives (Vamorolone)
  • Research into cardiac fibrosis in female carriers
Clinical Care & Access
  • DMD Care UK
  • DMD Hub UK
  • Improving access to clinical trials UK-wide
Technology & Quality of Life
How We Decide What to Fund

Every project we support is reviewed by an independent Scientific Advisory Board, made up of leading global experts in Duchenne.
This ensures:

  • Funding goes to the best science worldwide
  • No duplication of existing research
  • Every project has a clear path to delivery
  • Treatments move faster from lab to patient

We work closely with Duchenne UK to maximise impact and accelerate progress.

Transparency & Accountability

All funding decisions and investments are published in our Annual Accounts, available via the Charity Commission, covering every project supported since Alex’s Wish launched in 2013.