Diagnostics • September 17, 2025
Genomic Innovation to Support Newborn Screening of Rare DiseasesAlexion News Centre
Featured Articles
Clinical Trials • October 28, 2025
Shaping the future of R&D with real world insights into rare disease
Thought Leadership • June 11, 2025
Paving Regulatory Pathways in Rare Disease
Media [News] • April 10, 2025
Advancing Rare Disease Research Through Patient-Centric Trial Design
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.