Our Inspiration
- Living With Rare Diseases
- Real Stories
  - NF1
  - aHUS
  - gMG
  - HPP
  - LAL-D
  - NMOSD
  - PNH
- Support Services
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
- Conditions We Treat
  - aHUS
  - gMG
  - HPP
  - LAL-D
  - NF1 PN
  - NMOSD
  - PNH
- Medicines
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.
Our Research
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.
Our Commitment
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.
Our Company
Roberta is our inspiration

Despite having a constant struggle with symptoms, Roberta revisited her dreams and put herself through nursing school.
Your Career
- Career Opportunities
  - Join Our Team
- Working Here
  - Benefits
  - Culture
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.

General Inquiries

FIRST NAME

LAST NAME

COUNTRY

AREA OF INTEREST

INQUIRY

I'M NOT A ROBOT