Number of children on clinical trials doubles at Great Ormond Street Hospital, following funding through the DMD Hub

Duchenne UK is pleased to share a report from Great Ormond Street about the positive impact of the clinical staff posts we fund at the hospital.

The report highlights that the funding from the DMD Hub has provided Professor Franceso Muntoni’s neuromuscular research team with the resources they needed to develop, expand and stabilise.

Since the funding was agreed, the number of patients recruited into trials has increased from 139 in 2016/17 to 351 in 2017/18. 

In addition, at least 10 new commercial studies have started in that time.

The report says:

“Associated improvements in the organisational, training and administrative process of clinical trials conducted by the team has led to an improved patient and family experience - assessments take less time, waiting times are minimised and there are fewer cancellations.

The improvements in the team have also given the team the capacity to support and coordinate multicentre trials for potential new therapies, maximising access to these treatments.” 

Read the full report from GOSH here

These posts were funded by Duchenne UK, Harrisons Fund, Duchenne Research Fund and the Great Ormond Street Charity

Make sure you have JOINED THE HUB to stay informed about the latest trials and keep up-to-date with the latest news from the DMD Hub.

Visit the CLINICAL TRIAL FINDER to search for clinical trials.



What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. 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 DUCH10 £10 to 70070

For more information and interview requests

Visit www.duchenneuk.org

Molly Hunt – Communications Manger, Duchenne UK E: [email protected]


Published on 12 February 2018

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