Woman working in lab
Announcement

The FDA approved our long-acting C5 complement inhibitor for adults with generalized myasthenia gravis. Read more about our latest approval.

Inspired Every Day

The work we do is guided by people living with rare and devastating diseases. We are driven to continuously innovate and create meaningful value in all we do to help patients and families fully live their best lives.

SEE THEIR STORIES
  • Tanner, diagnosed with HPP at 4 days old, with a stuffed animal

    My hope for Tanner in the future is that he enters into adulthood. I kind of hope that he will be a doctor and maybe help kids like doctors have helped him.”

    RENE, MOM OF TANNER

    LIVING WITH HPP

    TANNER
    LIVING WITH HPP

  • Roberta, diagnosed with gMG at 16 years old

    This experience has taught me to look at the small things that count. Now, I feel that I have something to look forward to and I am who I want to be.”

    ROBERTA

    LIVING WITH gMG

    ROBERTA
    LIVING WITH GMG

  • The Trendys, diagnosed at 17, 14, 11, and 9 with LAL-D

    Now we knew what we were up against, we knew we could fight.”

    REBECCA, MOM OF TRENDY BROTHERS

    LIVING WITH LAL-D

    THE TRENDY FAMILY
    LIVING WITH LAL-D

  • Chelsey, living with NMOSD, painting

    I don't define myself as having a rare disease. It’s part of my life, but it’s certainly not who I am.”

    CHELSEY

    LIVING WITH NMOSD

    CHELSEY
    LIVING WITH NMOSD

  • Donnan, diagnosed with aHUS at 39 years old, and his son

    I can chase my kids and play again. I think knowing what I have brings a peace to my life.”

    DONNAN

    LIVING WITH aHUS

    DONNAN
    LIVING WITH aHUS

Every day, those who live with rare diseases and devastating conditions inspire us to push the boundaries of medicine, technology, and healthcare services. Our goal is to transform their reality for the better.

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Everyday Stories

Text: Patient Centricity in Rare Disease R&D

Pioneering transformational medicines for rare diseases requires not only scientific expertise but also a deep knowledge of the patient experience. To deliver medicines that truly meet the needs of patients and their families, we must listen, learn and incorporate their insights into our research and development (R&D).

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MORE NEWS

To commemorate our first anniversary as Alexion, AstraZeneca Rare Disease, patients and colleagues gathered across the globe to honor our legacy and celebrate our future in #RareDisease. #Alexion30

multiple images of people celebrating
There are over 7,000 known #RareDiseases in the world. Most of these diseases are genetic, and of those 70% start in childhood. For further advice and support: https://bddy.me/3GsrZBx
70% of genetic rare diseases start in childhood

In everything we do, we are empowered and committed to speak up and perform at our personal best to accelerate our collective impact for people living with #RareDiseases. Check out our career opportunities: https://bddy.me/3s0Ugce #hiring

multiple images of people working in a lab and in an office with text "#hiring"

Neuromyelitis optica spectrum disorder (#NMOSD) is a #RareDisease in which the immune system is inappropriately activated to target healthy tissues and cells in the central nervous system. For further advice and support: https://www.sumairafoundation.org/ 

Text: most people living with NMOSD experience unpredictable attacks, known as relapses. Each relapse can result in cumulative disability including vision loss, paralysis, and sometimes premature death

Gianluca Pirozzi, Head of Development & Safety, shares his journey and tips as the parent of a child with an ultra-rare disease in the latest issue of @RareRevolutionM focused on long-term caregiving. https://bit.ly/UnexpectedRAREJourney

Text: Quote from Gianluca Pirozzi
Jesse, diagnosed with gMG
I feel like tomorrow I can do something, whereas before, I didn’t feel like tomorrow was possible.”
JESSE LIVING WITH gMG