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.

Tanner, diagnosed with HPP at 4 days old, playing on a playground

TANNER
LIVING WITH HPP

REAL STORIES

Real Stories of Living With HPP

Adelyn

Diagnosed with HPP at 2 days old

Adelyn’s mother had a healthy pregnancy, but when Adelyn took her first breath, her entire chest cavity collapsed. She turned blue in color and her limbs were going the wrong way.

HEAR HER STORY

Evie

Diagnosed with HPP at 2 weeks old

When an ultrasound revealed that Evie had extremely fragile and transparent bones, doctors told expectant parents John and Lindsey that it was not likely their baby girl would live long after birth.

HEAR HER STORY

Tanner

Diagnosed with HPP at 4 days old

When Tanner was born, his mother, René, did not suspect anything was wrong with him. Then his doctors noticed that his wrists were retracting and he had an unusual amount of cartilage.

HEAR HIS STORY

Discover how we’re serving people living with HPP

HPP MEDICINE

See how we’re pushing the boundaries of HPP research every day

PIPELINE