Support from Alex’s Wish helps fund Duchenne Muscular Dystrophy (DMD) Clinical Trials Lectureship.

January 28, 2022 1:51 pm

We are delighted to report the outcomes of our grant for a Duchenne Muscular Dystrophy (DMD) Clinical Trials Lectureship. The grant, which was supported by a consortium of seven UK charities, saw Alex’s Wish joined by Duchenne UK, Action Duchenne, Duchenne Research Fund, Duchenne Now, Duchenne UK, Harrison’s Fund and Joining Jack to invest a total of £250,000.

Emma Hallam, Founder of Alex’s Wish says “It has been a real pleasure supporting Dr Michela Guglieri’s work over the past 5 years, and we are delighted that this role will continue to be funded by Newcastle University.  My son Alex has taken part in two clinical trials in Newcastle under Dr Guglieri’s team, and we see first-hand the amazing work they do and the impact they have on families affected by Duchenne. I would like to thank our fabulous supporters for their continued support and enabling us to support this vital work.”

The Clinical Trials Lectureship grant enabled Dr Michela Guglieri to act as the Clinical Research Team Leader within the John Walton Muscular Dystrophy Research Centre in Newcastle; a key role in one of the UK’s biggest Duchenne research centres, that ensures trials are run smoothly and safely, and generate good quality data.

Dr Guglieri comments “The project has supported the establishment of a strong clinical research team at the John Walton Muscular Dystrophy Research Centre and of national and international collaborations and networks which will play a key role in ensuring the clinical research in Duchenne Muscular Dystrophy will continue to come to the UK and to establish the UK as an expert and efficient country to deliver best care practice and clinical research.”

Since May 2017, as well as training and supporting the staff in the clinical research centre, over the course of the grant, Dr Guglieri has led 10 clinical trials in Duchenne, including:

  • The FOR DMD study aims to compare three different corticosteroid regimes, to determine which increases muscle strength the most in young people living with Duchenne, and which causes the fewest side effects. The results of this key trial will be published shortly – providing vital information for families and individuals deciding whether they or their son should take steroids.
  • The Vision DMD trial is testing the safety and effectiveness of Vamorolone – a potential drug that researchers hope could offer some of the effects of steroids with fewer side effects. Dr Guglieri was the study chair, supporting clinical sites who had medical or protocol queries and reviewing any adverse events seen during the trial to ensure patient safety.

The support from the 7 different charities has not only resulted in significant impact during the years of funding, but also a permanent position for Michela through Newcastle University. In this role, she will continue to work as Clinical Research Lead, supporting clinical research activities as well as national and international collaborations to improve care, treatment and research opportunities for people living with Duchenne muscular dystrophy.

Dr Guglieri continues “I aim to continue working on the Vision DMD and FOR DMD studies and publication plan with the grant support already in place. I am interested in establishing a DMD specific clinic with the aim to explore the feasibility of a multi-disciplinary clinic model and ultimately I aim to obtain a Professorship as a DMD clinical and clinical research expert.”

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