Rare diseases

Although individual diseases are rare, collectively, rare diseases are anything but – there are 7,000 different rare diseases and more than 400 million people suffering from them globally. Very few have an approved treatment available.

For those living with a rare disease, the impact is felt every day of a person's life. And although children make up around half of all those diagnosed, living with a rare disease requires life-long, often complex and challenging management.

But the number of people who feel the impact of rare diseases is even greater still. Because many rare diseases are genetic, it affects entire generations of families, many of whom become caregivers and advocates.

We develop innovative medicines and better diagnostics by exploring new ways to approach treatment. By changing medical practice, pursuing first or best-in-class medicines, focusing on cutting edge research and development and utilising new digital health technologies, we are helping to reshape the landscape of what it means to live with a rare disease.

There are still no treatments for 90% of rare diseases and so, research into better treatments, valuable therapies and more precise diagnosis will make a difference. Only through partnership with other companies, academic institutions and disease area experts can we make a positive difference to people living with a rare disease.

In rare disease, more than any other, we rely on close collaborations and co-creation with experts from the community from start to finish. Partnerships and trust with the rare disease community is paramount to our collective success. We are working with people with rare diseases at every stage to understand what is most meaningful to them so we can design our clinical trials and medicines with this in mind.

This dedication to partnership also extends beyond the patient community and into the scientific landscape. We recognise the value of external innovation and are dedicated to partnering with leading research and digital health companies to bring these to as many patients as possible.

By working together, we can build a better world for people with rare diseases.