Professor Francesco Muntoni awarded prestigious Novo Nordisk Prize

23 April 2026

Francesco Muntoni, one of our Research Vice Presidents, has been honoured with a prestigious Novo Nordisk award, at a ceremony in Denmark, in recognition of his pioneering contributions to the understanding and treatment of Duchenne muscular dystrophy (DMD).

A leading figure in neuromuscular research, Professor Muntoni has spent decades working to unravel the complexities of DMD and to develop therapies that could transform outcomes for people living with a muscle wasting condition and their families.

We spoke to him about his work, the challenges that remain, and what this recognition means for the field.

Fully understanding DMD to advance treatments

At the heart of Professor Muntoni’s research is a fundamental question: what happens in DMD, and how can it be managed?

“I am particularly interested in Duchenne, with the aim of better understanding what exactly happens in people with the condition – what the role of dystrophin is, and how we can restore it.”

Dystrophin is a protein essential for maintaining muscle strength. Its absence in DMD leads to progressive muscle loss. Understanding how dystrophin functions, and how to replace it, has been central to advancing treatments.

The biggest scientific hurdles

Despite decades of research, Professor Muntoni believes the world of muscle wasting conditions still holds many unanswered questions.

“Although neuromuscular conditions like Duchenne have been known for quite some time, there are still important gaps in our understanding of its biology. For example, we still don’t fully understand what dystrophin does in the brain and how this affects function.”

A breakthrough that changed the field

One of Professor Muntoni’s most significant contributions, recognised by the Novo Nordisk award, has been advancing the use of antisense oligonucleotides. Think of a gene like a long recipe. People with DMD have a typo in their recipe. An antisense oligonucleotide is like a small sticky note that covers the typo so the cook skips the mistake and can still make something usable.

“We recognised that dystrophin could be restored using antisense oligonucleotides. We were the first group in the UK to bring together a consortium to demonstrate this could be done safely and effectively.”

This work helped pave the way for the first FDA-approved therapy targeting DMD, marking a major milestone in the field.

What the Novo Nordisk award means

For Professor Muntoni, the recognition is both personal and symbolic.

“Of course, I feel honoured and pleased, it wasn’t something I expected. But more importantly, it recognises perseverance. In rare diseases, the first steps are crucial. They don’t solve everything, but they allow us to begin tackling the problem.”

He also highlights the broader significance:

“This is the first time the Novo Nordisk award has been given for a rare neuromuscular condition. It brings attention to the challenges patients and researchers face but also to the progress being made.”

The role of collaboration and support of our charity

Professor Muntoni emphasises that progress in DMD research has been a collective effort supported by charities and national collaboration.

“The early steps would not have happened without charity support in the UK. The first consortium we established, funded by the Department of Health, was coordinated with Muscular Dystrophy Campaign (now known as Muscular Dystrophy UK)”

He also highlights the importance of the NorthStar network:

“NorthStar has been crucial in helping us understand how DMD progresses over time. Years ago, we knew patients would get worse, but we didn’t have precise data on how. That information is essential for designing clinical trials and developing treatments.”

Looking ahead

While significant progress has been made, Professor Muntoni is clear that the journey is far from over. However, the recognition of his work and the field mark an important moment.

It signals not only how far DMD research has come, but also the growing global commitment to finding effective treatments for rare neuromuscular conditions.