Caring for the Caregivers: Mental Health in Rare Disease
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Article • May 11, 2026
Tina’s Big Trip: A rare disease story about going the extra mile
Tina’s Big Trip: A rare disease story about going the extra mile, is the newest title in Alexion, AstraZeneca Rare Disease’s Inspired By book series created to support the rare disease community, and especially families impacted by rare diseases. The story, which follows two sisters preparing for a journey to Italy with their father, reflects the daily resilience that families demonstrate while managing the unseen complexities of living with a rare disease.
Article • February 03, 2026
Advancing Policy, Partnerships and Progress for the Rare Disease Community
As we reflect ahead of Rare Disease Day, we reaffirm our commitment to pioneering innovations for the rare disease community and to shortening the time to diagnosis, improving access to lifesaving care, and advancing health care policies that support patients and families.
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.
Media [News] • April 10, 2025
Advancing Rare Disease Research Through Patient-Centric Trial Design
As Head of Development, Regulatory and Safety at Alexion, Gianluca Pirozzi channels his life’s experience with rare disease to inform his work at Alexion, ensuring clinical programmes are fueled by a patient-focused mindset.
Article • February 24, 2025
Supporting Patients on Rare Disease Day, and Every Day
Rare Disease Day recognizes the 400 million people worldwide – and 30 million in the United States – who are living with a rare disease. While significant progress has been made in understanding rare diseases and developing treatments, the need for continued awareness, research and support is as great as ever.
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 • 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.