Access to treatments

Key milestone: first UK patient enrolled in DMD gene therapy trial at DMD Hub site

12th May 2021

Pfizer have announced the enrolment of their first participant in a Duchenne muscular dystrophy (DMD) gene therapy trial in the UK. This is the first time a DMD patient in the UK has received gene therapy, and is a landmark moment in the mission to treat the condition.

The patient was enrolled at Newcastle, one of three locations in the UK where the DMD C3391003 Gene Therapy study is taking place. For more details on the trial in the UK, please consult the DMD Hub’s Clinical Trial Finder here.

The trial is a Phase 3 global, multicentre, randomised, double-blind, placebo-controlled study. In total, 99 boys across 55 sites in 15 countries will take part in the trial, with the first patient dosed in Barcelona in December 2020. It will evaluate the efficacy and safety of gene therapy construct PF-06939926, with participants being ambulatory boys between the ages of 4 and 7.

Dr Michela Guglieri, Consultant Neurologist at Newcastle Hospitals NHS Foundation Trust and UK Chief Investigator, said:

“The enrolment of the first UK patient in this Phase 3 gene therapy programme is a great achievement for the UK Duchenne community and we are very pleased to contribute to innovative research in the pursuit of future therapies. There are currently no approved disease-modifying treatment options available for all genetic forms of the disease in the UK. I’m very proud to be leading the UK arm of this global study.”

You can read the full press release from Pfizer here.

Published on 12 May 2021

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About Duchenne UK

Duchenne Muscular Dystrophy (DMD) is a devastating muscle-wasting disease. It is the most common and severe form of Muscular Dystrophy. Diagnosed in childhood, it mainly affects boys. There is currently no cure. Started by families affected by the disease, Duchenne UK has one clear aim – to end Duchenne.

Duchenne UK are funding research that’s focused on getting treatments to those affected now – as well as pushing for an effective treatment in the future.

Duchenne UK connects leading researchers with industry, the NHS and patients to challenge every stage of drug development, from research to clinical trials to drug approval. They connect families with each other to create a network of mutual support and to pool resources, knowledge and experience.

How to donate

COVID-19 has caused a funding crisis in medical research. But we cannot, and will not, let it slow us down in our mission to find new and effective treatments for DMD. Because this generation of boys cannot wait. Donate now, and together we will end Duchenne

Duchenne UK is entirely reliant on donations. This can be done via:

Direct Debit – Duchenne Direct
Individual Donation – Donate
If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
Take part in one of our fundraising events – Events
Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.