Our Disease Areas
We 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.
Expanding Our Leadership in Rare Disease
We have more than 30 years of rare disease leadership and 20-plus years of real-world evidence transforming the patient journey and establishing new standards of care. We remain committed to following the science, deepening disease understanding and enhancing outcomes for patients and caregivers.
Our specialized therapeutic expertise shapes the future treatment landscape, delivering meaningful advances in care while bringing patient voices directly into our development process to guide research priorities.
Combining our clinical and commercial expertise with AstraZeneca's global infrastructure, we're exploring new molecular targets and accelerating our pipeline. Through internal innovation and external partnerships, we pursue complementary therapeutic approaches to address significant unmet medical needs across our five disease areas of focus:
Rare Neurology
Rare Bone & Endocrine Disorders
Rare Cardiology & Amyloidosis
Rare Tumours
Our commitment
Deliver transformational therapies that advance rare disease research and create new treatment options for the communities we serve.
Innovation
Learn how we're advancing research to help address rare diseases with high unmet needs to help make a difference for patients and their families.
Veeva ID: GL/ALL/0105