Celebrating Professor Francesco Muntoni: advancing progress for the SMA community
At SMA UK, we are proud to celebrate Professor Francesco Muntoni’s recognition as the 2026 Novo Nordisk Prize laureate. The Novo Nordisk Prize is awarded annually to scientists whose work has significantly advanced medical science and improved people’s lives – a description that resonates profoundly with Professor Muntoni’s career and impact. While much of his pioneering research has focused on Duchenne muscular dystrophy, his impact reaches far beyond a single condition, helping to transform the outlook for people living with neuromuscular conditions, including spinal muscular atrophy (SMA).
For families affected by SMA, the past decade has marked a huge shift. Where once there were no treatments, today there are therapies that target the genetic cause of the condition. This progress has been made possible by decades of research into how we understand and intervene in genetic disease, a field Professor Muntoni has helped to shape.
From genetic discovery to real-world impact
Throughout his career, Professor Muntoni has worked at the forefront of translational medicine, bridging the gap between scientific discovery and meaningful treatments for patients.
His early work on RNA-based therapies, including antisense oligonucleotides (ASOs), demonstrated that it is possible to intervene at a molecular level to modify genetic disease. This approach (correcting how genes are read and expressed) underpins several of the therapies now transforming outcomes for people with SMA.
For the SMA community, these scientific advances have been life-changing. Treatments that increase production of the survival motor neuron (SMN) protein (the protein missing in SMA) are now improving survival, motor function, and quality of life for many individuals.
While these breakthroughs are the result of global collaboration, Professor Muntoni’s work has played an important role in building the scientific and clinical foundations that made them possible.
A leader in neuromuscular care in the UK
Since moving to the UK, Professor Muntoni has established one of Europe’s leading paediatric neuromuscular centres at University College London and Great Ormond Street Hospital. This centre has become a vital hub for diagnosis, care, and research across a wide range of conditions, including SMA.
Through this work, thousands of children and families have benefited from earlier diagnosis, improved standards of care, and access to clinical trials.
His leadership has also helped to strengthen the UK’s role in international research, ensuring that people living with SMA have opportunities to participate in studies that drive forward new treatments and a deeper understanding of the condition.
Driving collaboration and progress
Progress in SMA has only been possible through collaboration, between researchers, clinicians, charities, industry, and, crucially, people living with the condition and their families.
Professor Muntoni has long championed this collective approach. By bringing together expertise across disciplines and borders, he has helped create the systems needed to turn scientific breakthroughs into real-world treatments.
For the SMA community, this has meant faster progress, stronger research networks, and a growing pipeline of potential therapies.
Balancing hope with realism
As new treatments continue to emerge, Professor Muntoni has consistently emphasised the importance of combining hope with scientific rigour.
For families affected by SMA, this balance is essential. While today’s therapies are transforming lives, there is still more to do, from improving access and long-term outcomes to developing next-generation treatments.
His commitment to evidence-based progress reflects a shared priority across the SMA community: to ensure that advances are meaningful, accessible, and grounded in real-world benefit.
Looking ahead
Giles Lomax, CEO said, “Professor Muntoni’s work represents the kind of scientific progress that has fundamentally changed what is possible for people living with SMA. At SMA UK, we’ve seen firsthand how advances in genetic medicine are transforming lives offering hope where there once was very little. This recognition is not only well deserved, but a powerful reminder of the importance of continued investment in research, collaboration, and the voices of the SMA community.”
At SMA UK, we remain committed to supporting research, improving care, and amplifying the voices of people affected by SMA. The foundations laid by leaders like Professor Muntoni continue to drive us forward.
As we look to the future, we do so with cautious optimism and with determination to ensure that everyone affected by SMA can benefit from the advances still to come.
