We are pioneering new possibilities for the rare disease community.
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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.
Article • November 12, 2026
From the Marines to Rare Disease: How One Veteran Found His Next Mission at Alexion
Article • November 11, 2026
Alexion Opens STEM Learning Lab to Inspire Future Innovators
Article • September 04, 2026
Advancing Newborn Screening Through Earlier Access to Diagnostic Testing
Article • May 18, 2026
Across the United States, millions of families are navigating the complexities of rare disease. Behind many of these journeys is a caregiver coordinating appointments, advocating for access and care, balancing responsibilities at home and work, and carrying a significant emotional load.<br>Recognizing the toll these challenges can have on families, Alexion, AstraZeneca Rare Disease collaborated with caregivers and mental health professionals to develop More Than Rare, a three-part webinar series focused on patient and caregiver mental health.
Article • May 11, 2026
Tina’s Big Trip: A rare disease story about going the extra mile, is the newest title in Alexion, AstraZeneca Rare Disease’s Inspired By book series created to support the rare disease community, and especially families impacted by rare diseases. The story, which follows two sisters preparing for a journey to Italy with their father, reflects the daily resilience that families demonstrate while managing the unseen complexities of living with a rare disease.
Article • February 03, 2026
As we reflect ahead of Rare Disease Day, we reaffirm our commitment to pioneering innovations for the rare disease community and to shortening the time to diagnosis, improving access to lifesaving care, and advancing health care policies that support patients and families.
Clinical Trials • October 28, 2025
Studying a rare disease often involves working with fragmented information, much like piecing together scattered pages of different books, each offering part of the story but rarely forming a complete picture. Researchers face this challenge due to small, often underdiagnosed, patient populations and limited understanding of how these diseases progress over time.
Diagnostics • September 17, 2025
Today, while more than 10,000 rare diseases are known, recommended newborn screening panels cover only about 40 core conditions. This gap underscores the need for innovative approaches to expand the reach of newborn screening.
Thought Leadership • June 11, 2025
Many rare disease medicines are often a first of their kind, and a clear roadmap for their development and approval does not exist. Navigating the complex and sometimes unprecedented regulatory environment for rare disease requires innovation, collaboration and a patient-first mindset.
Media [News] • April 10, 2025
As Head of Development, Regulatory and Safety at Alexion, Gianluca Pirozzi channels his life’s experience with rare disease to inform his work at Alexion, ensuring clinical programmes are fueled by a patient-focused mindset.
Article • February 24, 2025
Rare Disease Day recognizes the 400 million people worldwide – and 30 million in the United States – who are living with a rare disease. While significant progress has been made in understanding rare diseases and developing treatments, the need for continued awareness, research and support is as great as ever.
Patient Support • February 05, 2025
We spoke with Cristina Quarta, Executive Medical Director, about a group of rare diseases known as amyloidosis, the medical needs within this community and how her work as a cardiologist shapes her approach to research and development (R&D) for rare cardiac conditions.
Patient Support • September 23, 2024
With 10,000 known rare diseases defined today, the path to diagnosis can often resemble a puzzle, with physicians as detectives searching for clues. Many physicians never encounter a rare disease beyond the pages of their medical school textbooks.
Research and Development • September 18, 2024
To create meaningful advances in rare disease research, it takes an innovative culture, strong collaboration, and a patient-centric vision for delivering transformational solutions. At Alexion, these pillars are important to successfully develop impactful medicines for patients.
Patient Support • June 25, 2024
Artificial Intelligence (AI) is rapidly advancing and has the potential to have a transformative impact on clinical research and development (R&D).
Research and Development • June 08, 2024
There is a sense of urgency that underlies research in rare diseases, because these diseases are often rapidly progressive.
Patient Support • April 18, 2024
The voices and perspectives of patients play an essential role in rare disease research and development (R&D).
Patient Support • April 16, 2024
Diagnostics and artificial intelligence (AI) systems are transforming research and development (R&D) in the healthcare industry.
Health Equity • February 06, 2024
Collectively, more than 400 million people around the world are living with a rare disease, defined as conditions that affect a small number of people compared to the general population.
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.