Research and Development • July 16, 2026
Advancing genomic medicine in rare diseasesAlexion 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
Health Equity • May 21, 2026
Understanding treatment impact and addressing disparities in rare disease care
Article • May 11, 2026
Tina’s Big Trip
Article • February 03, 2026
Pioneering new possibilities for the rare disease community
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).
Research and Development • June 08, 2024
Getting to know Seng Cheng, an Alexion R&D Leading Voice
There is a sense of urgency that underlies research in rare diseases, because these diseases are often rapidly progressive.
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.