Paving the way to new clinical trials

Published Date
Alexa Follen

Muscular Dystrophy UK is delighted to be working with fellow charities to make a £1.2m investment in roles to support clinical trials.

Announced in December 2015, this money is supporting 16 new roles, paving the way for more clinical trials. Muscular Dystrophy UK is funding four of these new roles.

All 16 roles are supporting people with Duchenne muscular dystrophy, with two of these – Muscular Dystrophy UK-funded roles at Great Ormond Street Hospital – also covering other neuromuscular conditions.

The initiative could help people like five-year-old Shiv (pictured), who has Duchenne muscular dystrophy.

Today we can announce that 10 of these posts have now been recruited to support new clinical trials in the UK.

Why is this investment so important?

Research into muscle-wasting conditions is at an exciting stage. Some approaches to treatment are in late stage clinical trial and one drug – Translarna, to treat nonsense mutation in Duchenne muscular dystrophy – is now an approved drug in England and Northern Ireland.

However, at a meeting in Newcastle in July 2015, parents Emily Crossley and Alex Johnson – both of whom have sons affected by Duchenne muscular dystrophy – gave alarming reports that Muscle Centres were turning down clinical trials for Duchenne: not because the trials lacked potential, but because they didn’t have the staffing and capacity to take them on. This was confirmed by leading clinicians at UK Muscle Centres.

This risked creating a major blockage, and holding up promising advances in research into potential treatments for Duchenne muscular dystrophy.

A November 2015 Muscular Dystrophy UK audit of clinical trial capacity confirmed this picture, and also demonstrated it was a major issue for trials into other types of muscle-wasting condition, such as spinal muscular atrophy (SMA).

You can read other key findings from the audit here.

Who is funding these new roles and where are they based?

Muscular Dystrophy UK has put £360,000 into funding four new roles at Great Ormond Street Hospital, John Walton Muscular Dystrophy Research Centre in Newcastle and Alder Hey Children’s Hospital in Liverpool.

Supporting 12 other vital roles are: Action Duchenne, Alex’s Wish, Duchenne Now, Duchenne Research Fund, Duchenne UK, Great Ormond Street Children’s Charity, Harrison’s Fund and Joining Jack.

Muscular Dystrophy UK is committed to accelerating the development of effective new drugs through clinical trials, and we are delighted to be working in partnership with fellow charities to help make this happen.

We would like to thank the families whose fundraising has made our £360,000 investment possible – and thank all those whose efforts have led to the overall £1.2m investment.

Below are updates on all 16 roles:

The John Walton Muscular Dystrophy Research Centre at Newcastle University

A Clinical Trial Co-ordinator is now in post, and a Research Physiotherapist has been appointed and is due to start very shortly. An administrative post, a nurse and Clinical Research Associate are all close to appointment.

Alder Hey Children’s Hospital in Liverpool

One Research Physiotherapist is in post and the roles of Trials Co-ordinator and Clinical Fellow are now recruiting.

Great Ormond Street Hospital in London

These posts are being funded in collaboration with the NHS Trust and GOSH charity

Two Clinical Research Fellows have been appointed, with one more close to recruitment. Two Research Physiotherapists posts (made up of one full-time and two part-time), one Physiotherapy Assistant and a Clinical Trial Co-ordinator have also been appointed, along with a Speciality Doctor and Research Nurse.  A senior lecturer is close to appointment, with a Professor and Data Manager still to come.

University Hospitals Bristol NHS Foundation Trust

A Clinical Research Physiotherapist will be recruited very soon.

Muscular Dystrophy UK’s impact on Duchenne research

In addition to the work to increase clinical trial capacity, Muscular Dystrophy UK has been been contributing to Duchenne research by:

  • funding Clinical Trial Co-ordinators over five years investing £400,000
  • providing support for over 20 Clinical Research and Training Fellows, investing more than £3m over 20 years
  • initiating and investing £1m in the North Star project, launched in 2006, to improve care standards and find ways to test the benefits of drugs in clinical trials.

We have a shared vision of working with families to focus on increasing clinical trial capacity for everyone living with a muscle-wasting condition.

For more information, please contact Peter Sutton on


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