In select countries, third-party companies manage our local commercial operations.
Albie, one of our guiding stars
When Albie was born, his mom, Charlotte, said that he was “absolutely fine. Everything was fine for about two weeks.”
Our medicines help people like Donnan
As a father of two young children, Donnan recalls being afraid of what the future would hold prior to his diagnosis.
We innovate for Ruthie
Married with a newborn son, Ruthie was scared to learn that PNH was a life-threatening illness that at the time had no approved treatment.
We give back for families like Evie's
At only three months old, Evie's parents were told that her case was severe and that there were no approved or effective treatment options.
Roberta is our inspiration
Despite having a constant struggle with symptoms, Roberta revisited her dreams and put herself through nursing school.
Make a difference for families like Tristan's
Tristan's parents experienced a whirlwind of emotions and fear after learning their 5-year-old son had such a rare and serious disease.