Article • September 22, 2026
How can data, healthcare access and global collaboration advance health equity in rare diseases?
Clinical Trials • October 15, 2026
Shaping the future of R&D with real world insights into rare disease
Article • September 22, 2026
How can data, healthcare access and global collaboration advance health equity in rare diseases?
Thought Leadership • August 19, 2026
Advancing Rare Disease Research Through Patient-Centric Trial Design: Hear from Alexion R&D leader Gianluca Pirozzi
Data Science and AI • August 13, 2026
AI to shorten the rare disease diagnosis
Thought Leadership • August 12, 2026
Keeping Patients at the Forefront of Rare Disease Innovation: Hear from Alexion R&D leader Seng H. Cheng
Research and Development • August 12, 2026
Building on a legacy in complement science, we are advancing a new era of medicines focused on precision, specificity and improved outcomes for patients.
Health Equity • May 21, 2026
Alexion’s leadership in rare disease is driven by the many individuals whose expertise, creativity and tenacity fuel our commitment to advancing innovative science.
Article • May 11, 2026
Tina’s Big Trip is a heartfelt book that follow two sisters preparing for a journey to Italy as their family navigates the unseen complexities of rare disease.
Article • February 03, 2026
Rare Disease Day unites communities to drive strategies, funding, diagnosis and treatment, connecting local and global efforts for earlier, equitable care.
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.
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 • 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).
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.
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.