Our rare disease team works to develop effective therapies for patients affected by rare disorders, which are defined by the EU as those diseases affecting fewer than 1 in 2000 patients.
Over the past decade, scientific advances have increased understanding of rare diseases and their underlying causes, enabling the development of critical treatment options for some.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Knowledge Center ▸ Endocrinology Knowledge Center ▸