Thought Leadership • June 11, 2025
Paving Regulatory Pathways in Rare DiseaseAlexion News Centre
Featured Articles
Diagnostics • September 17, 2025
Genomic Innovation to Support Newborn Screening of Rare Diseases
Media [News] • April 10, 2025
Advancing Rare Disease Research Through Patient-Centric Trial Design
Article • February 24, 2025
Supporting Patients on Rare Disease Day, and Every Day
Patient Support • February 05, 2025
Addressing the Unmet Needs of the Amyloidosis Community
Patient Support • September 23, 2024
Shortening the Path to Diagnosis in Rare Disease
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
Getting to know Nick France, an Alexion R&D Leading Voice
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
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.
Patient Support • November 08, 2023
Getting to Know Mary Kunjappu, an Alexion R&D Leading Voice
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
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.
Clinical Trials • November 01, 2023
Getting to know Adrian Kielhorn, an Alexion R&D Leading Voice
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
Children’s Book Series is Inspired By Patients Living with Rare Diseases
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.