Rare Diseases
Advancing Rare Disease Care
Shionogi is committed to addressing the significant unmet needs of people living with rare diseases, their families and caregivers.
Rare diseases are often serious, progressive conditions that can have a profound impact on patients and those around them. Many people face delayed rare disease diagnosis, limited treatment options and challenges accessing appropriate treatment, care and support.
Building on our heritage in addressing areas of high unmet medical need, Shionogi is expanding its focus in rare diseases through scientific innovation, investment in areas of greater patient need, and long-term collaboration. Our growing commitment spans rare neurological and paediatric diseases, including Amyotrophic Lateral Sclerosis, (ALS), Fragile X syndrome, Pompe disease and other rare conditions where patients and families continue to face gaps in diagnosis, treatment and care.
At Shionogi, we believe listening is a vital part of patient-centred care. Our patient-first approach is grounded in the belief that better solutions are created with patients, not for them. By listening to and learning from the lived experiences of patients and caregivers, we seek to build deeper understanding, and ensure patient perspectives help shape our activities and decision-making.
We recognise that sustainable progress in rare diseases requires more than developing new medicines. Through partnerships and engagement with patient communities and organisations including EURORDIS and EUPATI, we work alongside patients, caregivers, healthcare professionals, researchers, advocacy organisations and policymakers to help strengthen the rare disease ecosystem across Europe and support health equity.
By combining scientific innovation with patient partnerships and cross-sector collaboration, we aim to advance equitable access to care, improve outcomes and help create a future where people affected by rare diseases receive earlier diagnosis, effective treatment and the support they need throughout their journey.