Our Therapy Areas
We look to apply our rare disease expertise where it's needed most, and where we can make the biggest impact for people living with rare diseases. This often means we are exploring disease states where scientific progress has stalled for decades, and where patients have limited—or zero—treatment options.
Bone Metabolism
Researching and developing innovative treatments that aim to target the underlying causes of rare bone disorders.
Cardiology
Following the science to develop and evaluate potential innovative treatment options to help people living with rare cardiology conditions.
Endocrinology
Identifying potential new opportunities to advance care for those impacted by rare endocrine diseases.
Hematology
Working to improve potential outcomes for more people living with rare hematological diseases worldwide.
Nephrology
Enhancing the understanding of rare renal diseases and accelerating the development of potential therapies.
Neurology
Striving to advance scientific understanding in rare neurology and to accelerate the development of potential life-changing treatments.
Rare Cancers
Striving to advance scientific understanding of rare cancers to bring new solutions.