Pioneering rare disease treatments - Ipsen

Rare Disease

Rare diseases impact one in 10 people. Globally, that is 350-400 million people living with one of over 7,000 identified rare diseases.​

WHO WE ARE

We are Ipsen in rare disease. We are ordinary people with the extraordinary ambition to improve the health and prolong the lives of people living with rare diseases.

At Ipsen, we embrace the challenge of bringing to market medicines for people living with a rare disease because we are here to stand up for those who often get left behind.

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We are Ipsen in Rare Disease

We focus our research and innovation on the greatest areas of unmet need and where we can make a meaningful impact for patients. Our current priority is advancing medicines and scientific understanding in rare liver disease, rare bone disease and endocrine growth disorders.

Ipsen’s unwavering commitment to patients​

People living with a rare disease face daily barriers, including delayed diagnoses and delayed or inadequate treatment. The chronic, progressive, degenerative, and frequently life shortening nature of many rare diseases can have a profound impact on quality of life. We’re committed to supporting patients from diagnosis to treatment and partnering with the community to deliver better outcomes for everyone. ​

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Our focus on science​

One of the biggest challenges in rare disease is the limited scientific research and knowledge available to inform our understanding. We initiate clinical development programs that advance scientific understanding of rare diseases and their impact, to pave the way for better disease management and future therapeutic advances.

Clinical trials

Our focus on innovation

Our mission is to bridge the gap between innovative science and improved outcomes for patients. We are focused on new treatment options that address what matters to doctors and patients and are investing our time and resources into investigating medicines that work differently, that have the life-changing potential to alter or slow the course of a disease and provide relief from the pain and distress of debilitating symptoms.

Our pipeline

Top stories in rare disease

23 May 2023 1 mins read

Fireside Chat: Innovation and Policy Change in Rare Diseases in Europe 

The rare disease community continues to strive for innovative solutions to meet patient needs and ensure no person living with a rare disease is left…

12 May 2023 4 mins read

Understanding the true impact of living with FOP

This year marks the 17th anniversary since the discovery of the ACVR1 gene was announced, a major scientific milestone in fibrodysplasia ossificans progressiva (FOP) research that helped us…

20 December 2022 1 mins read

Living with PBC: The Person Behind the Chart

‘Invisible’ diseases like primary biliary cholangitis (PBC) can be difficult to recognise, diagnose and to manage effectively. Here Prof. Gideon Hirschfield (University of Toronto, Departments…

03 November 2022 4 mins read

A Life in a Day with FOP: from Empathy to Understanding

You can never truly understand what it’s like to live with an ultra-rare condition unless you’ve experienced it for yourself. And with this in mind,…

05 September 2022 5 mins read

‘A Life in a Day’: the realities of living with FOP

I was honored to be invited to take on the challenge to live ‘A Life in a Day of a patient with FOP’ – but…

05 August 2022 3 mins read

ILC 2022: Let’s talk PBC

The International Liver Congress (ILC)1 provides a key opportunity for us to meet and discuss the management and research of liver diseases. Given the online…