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-
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 -
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 -
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 -
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 -
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.
5
APPROVED MEDICINES7
RARE DISEASES & DEVASTATING CONDITIONS25+
OFFICES WORLDWIDE3000+
TALENTED COLLEAGUESEveryday Stories

Over the past year, we marked 30 years of leadership in rare diseases by celebrating our pioneering legacy and unyielding commitment to patients. But there is much more work to be done to support rare disease communities across the world, and as we look toward the next 30 years, we will continue to lead the way.
Learn More
I feel like tomorrow I can do something, whereas before, I didn’t feel like tomorrow was possible.”JESSE LIVING WITH gMG