We are pioneering new possibilities for the rare disease community.
Medicines approved
Explore Our MedicinesRare diseases & conditions we treat
Conditions We TreatYears of pioneering science
Our Approach
There are over 400 million people living with a rare disease. Discover their personal stories and experiences as they share their journey with us.
Our research and development is fueled by a relentless sense of urgency and an steadfast commitment to helping more rare disease patients and their families around the world.
Learn how we are addressing some of today’s most pressing rare disease challenges by investigating in new targets, modalities and technologies, with more opportunities for innovation than ever before.
Patient Support • June 25, 2024
Getting to know Chathuri Daluwatte, an Alexion R&D Leading Voice
Research and Development • June 08, 2024
Getting to know Seng Cheng, an Alexion R&D Leading Voice
Patient Support • April 18, 2024
Getting to know Sophia Zilber, Associate Director, Statistical Programming
Patient Support • April 16, 2024
Getting to know Banu Sankaran, an Alexion R&D Leading Voice
Health Equity • February 06, 2024
Driving health equity for the rare disease community
Patient Support • November 08, 2023
Science is hard, but rare disease research and development (R&D) stands apart. Because individual patient populations are small and many rare diseases are not well understood, research in this space can often bring added complexity.
Clinical Trials • November 07, 2023
Why is conducting clinical trials for rare diseases difficult? Because studying a rare disease poses unique challenges and requires thinking differently.
Clinical Trials • November 01, 2023
Alexion’s leadership in rare disease is driven by the many individuals whose expertise, creativity and tenacity fuel our commitment to advancing innovative science.
Patient Support • June 30, 2023
At Alexion, our mission is to transform the lives of people affected by rare diseases and one of the tangible ways we raise awareness is by developing tools and resources to educate about the impact of rare diseases on patients, caregivers, and families.