Data Science and AI • August 13, 2026
AI to shorten the rare disease diagnosisAlexion News Centre
Featured Articles
Thought Leadership • August 19, 2026
Advancing Rare Disease Research Through Patient-Centric Trial Design: Hear from Alexion R&D leader Gianluca Pirozzi
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
Following the Science: The Evolution of Complement Inhibition in Rare Disease
Research and Development • July 16, 2026
Advancing genomic medicine in rare diseases
Health Equity • May 21, 2026
Understanding treatment impact and addressing disparities in rare disease care
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
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
Pioneering new possibilities for the rare disease community
Rare Disease Day unites communities to drive strategies, funding, diagnosis and treatment, connecting local and global efforts for earlier, equitable care.
Clinical Trials • October 28, 2025
Shaping the future of R&D with real world insights into rare disease
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
Genomic Innovation to Support Newborn Screening of Rare Diseases
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
Paving Regulatory Pathways in Rare Disease
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.
Patient Support • February 05, 2025
Addressing the Unmet Needs of the Amyloidosis Community
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
Getting to know Chathuri Daluwatte, an Alexion R&D Leading Voice
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
Getting to know Sophia Zilber, Associate Director, Statistical Programming
The voices and perspectives of patients play an essential role in rare disease research and development (R&D).
Patient Support • April 16, 2024
Getting to know Banu Sankaran, an Alexion R&D Leading Voice
Diagnostics and artificial intelligence (AI) systems are transforming research and development (R&D) in the healthcare industry.
Health Equity • February 06, 2024
Driving health equity for the rare disease community
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
Understanding & Overcoming the Challenges in Rare Disease Clinical Trials
Why is conducting clinical trials for rare diseases difficult? Because studying a rare disease poses unique challenges and requires thinking differently.