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

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, which ensures trials are run smoothly and safely and generate good quality data.

Dr Guglieri says: “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 DMD 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 seven 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.

“I aim to continue working on the Vision DMD and FOR DMD studies and publication plan with the grant support already in place,” adds Dr Guglieri. “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.”

“We are so pleased to have been able to fund Dr Michela Guglieri’s work during the last five years,” says DRF director of operations Sheli Rodney. “She is a driving force in the Duchenne arena, working tirelessly with families, colleagues and charities to progress care and research. We are delighted that she has been offered a permanent position to continue in her role as clinical research lead, and we look forward to many more years of collaboration with her.”

Click here to learn more about this project. 

Quotes from partner charities:

“Action Duchenne are proud to have stood shoulder to shoulder with these 6 other charities; investing as a group to make this project possible. The impact that this project has had on advancing Duchenne research, clinical trials, and enabling a leading light in the international Duchenne community comes as a result of us working in partnership; truly demonstrating the power of being ‘stronger together’. Action Duchenne wishes to thank each of the partner organisations, along with Michela herself for her hard work and dedication to the Duchenne community. We are focussed on a more collaborative approach in order to best serve the Duchenne community and will relish the opportunity to partner with these organisations to deliver future projects.” – Florence Boulton, national director, Action Duchenne

“We are delighted with the impact that Dr Michela Guglieri has been able to make during the last 5 years of our grant. Her leadership, knowledge and expertise have driven forward advances in clinical trials and care for everyone living with Duchenne muscular dystrophy. We are always grateful for the unwavering dedication she shows to helping our community. Duchenne UK and Joining Jack are looking forward to continuing working with her on projects moving forward.” – Emily Crossley & Alex Johnson, co-founders, Duchenne UK

“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.” – Emma Hallam, founder, Alex’s Wish

“We have been delighted with every effort and outcome achieved by Dr Micheal Guglieri over the five years of our funding. Families and patients living with Duchenne Muscular Dystrophy as well as Harrison’s Fund are indebted to her dedication to the disease, her passion to advance therapies and clinical trials and her continued support of the wider community. We look forward to supporting further in the future.” – Alex Smith, founder, Harrison’s Fund