Driving the research strategy and project pipeline with our new Scientific Advisory Board

By Dr Alessandra Gaeta, Director of Research & Development, Duchenne UK

I am delighted to announce our new Scientific Advisory Board (SAB), which includes some of the world’s experts in Duchenne Muscular dystrophy (DMD), with a wide range of scientific expertise, knowledge and experience in research, clinical practice, and drug discovery and development.

With several new therapies for DMD being approved around the world, and new exciting progress being made in DMD research and development (see my recent World Muscle Congress update here for examples of these), we want to continue to fund innovative and impactful research to accelerate transformative treatments to the clinic, advance knowledge in the field and ultimately improve the lives of people with DMD. The SAB will be crucial to this.

I am delighted that the following people are now members of the Duchenne UK’s SAB.

Professor Giovanni Baranello, Professor of Paediatric Neuromuscular Disorders in the Developmental Neurosciences Department at the University College London and Great Ormond Street Hospital Institute of Child Health.

Dr John Bourke, Consultant cardiologist (Electrophysiologist) at the Department of Cardiology, Freeman Hospital, Newcastle upon Tyne, UK, and Senior Clinical Lecture at Institute of Translational and Clinical Research, Newcastle University.

Professor Dame Kay Davies, Co-founder and a director of the Oxford Centre of Gene Function, and co-director of the MDUK Oxford Neuromuscular Centre.

Professor Jordi Diaz Manera, Professor of Neuromuscular Diseases, Translational Medicine and Genetics at John Walton Muscular Dystrophy Research Center Life, at the University of Newcastle.

Dr Tina Duong, Director of Clinical Outcomes Research and Development at Stanford University Neuromuscular division.

Dr Dada Pisconti, Associate Professor in the department of Biochemistry and Cell Biology at SUNY Stony Brook, New York.

Dr Isabelle Richard, Director at the Centre National de la Recherche Scientifique (CNRS), France, and who is heading the muscular dystrophies team within the INSERM U951 in Genethon, Evry, France.

Dr Valeria Ricotti, Honorary clinical lecturer in the Developmental Neurosciences Department at the University College London and Great Ormond Street Hospital Institute of Child Health.

Dr Carina Schey, Consultant health economist with a doctorate focusing on assessing the value-add of orphan drugs, awarded by the University of Groningen in the Netherlands.

Professor Francesco Saverio Tedesco, Professor of Neuromuscular Biology and Regenerative Medicine at University College London, Senior Group Leader at the Francis Crick Institute and Honorary Consultant Paediatric Neurologist at Great Ormond Street Hospital.

Dr Graeme Wilkinson, Head of Virtual Research and Development at the Medicines Discovery Catapult.

Why they have joined our new Scientific Advisory Board 

I would like to share what some of the new board members have to say about their involvement and why they joined.

Dr John Bourke

“As a cardiologist, I was delighted to be invited to become a member of Duchenne UK’s Scientific Advisory Board. I will use the opportunity to help Duchenne UK plan its portfolio of research for the next three years, focusing projects on the newer insights and promising novel therapies that are becoming available, while continuing to support improvements in the delivery of cardiac care more generally.”

Professor Francesco Saverio Tedesco

“I am thrilled to join the Scientific Advisory Board of Duchenne UK. I hope to be of help for the DMD community during these exciting times of rapidly evolving therapeutic opportunities for neuromuscular disorders.”

Dr Dada Pisconti

“It's been the greatest honour of my life to know that the two most amazing women I have ever met, Alex and Emily, trusted my opinion. I was humbled, honoured and a bit scared because it's a big responsibility.”

Professor Dame Kay Davies

“I am delighted to be part of the Scientific Advisory Board of Duchenne UK. Duchenne UK has a strong reputation for playing a key role in supporting basic research and translation into the clinic for the development of an effective treatment for DMD boys. Their advocacy has also been instrumental in ensuring treatments are accessible as soon as possible.”

Dr Carina Schey

“I joined the board because I believe that my expertise as a health economist with a special interest in rare diseases, and willingness to help could make a difference to patients with DMD, as well as their families. It's that simple. If every human being in society contributed a bit of their expertise and time to worthy causes, the world would be in a significantly better state.”

Dr Tina Doung

“Joining Duchenne UK's Scientific Advisory Board, my passion is fueled by the belief that we can alleviate the burden of Duchenne muscular dystrophy through a multifaceted approach. With each step, I am dedicated to pioneering novel approaches that not only measure function but also empower autonomy. In this role, I see this as a personal commitment to making a meaningful impact by being a catalyst for positive change, where breakthroughs are not just data points but stories of improved lives. In research and development, I hope to turn inspiration into tangible outcomes. It is such an honor to be a part of Duchenne UK’s scientific advisory board to be part of a collective force working towards a future where each discovery is a testament to the potential we hold to transform lives.”

Dr Valeria Ricotti

“Duchenne muscular dystrophy remains one of the most challenging paediatric diseases. To change its fate has been a purpose very close to my heart. I feel honoured to be part of the Duchenne UK community and work together in peruse of our common goal.”

Shaping Duchenne UK research strategy

We will be meeting with the SAB in November to discuss and develop our research strategy.

I am very grateful for the SAB members’ time and contributions, and I look forward to working with them and to share with you the new research strategy in the new year.

Our commitment to impactful research continues

With so many new opportunities on the horizon, it’s crucial that we are strategic about where we invest the money that our donors work very hard to raise to support us.

More than ever, we are committed to continue to fund the best research that can be most impactful for people living with DMD, and we are grateful for the ongoing support of our partners Alex’s Wish, Joining Jack, and Duchenne Research Fund, the Family and Friends Funds, and everyone who takes on a challenge, such as the Duchenne Dash to running a marathon, to help us achieve this.

Published on 29 October 2023

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