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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.

REAL STORIES

Living Their Lives Every Day

We know that beginning with their first symptoms through an often long path to diagnosis and treatment, people living with a rare disease have to navigate a sobering new normal while managing other life priorities.

But rare disease patients persevere. With our help, they’re reconnecting, moving forward, and making every day of their life what they want it to be.

FILTER

Conditions

All conditions

NF1

aHUS

LAL-D

gMG

NMOSD

HPP

PNH

KENDALL
LIVING WITH NF1

Albie, diagnosed with LAL-D as an infant, with his mother

ALBIE
LIVING WITH LAL-D

Joe, diagnosed with PNH at 24 years old, with his fiancée and child

JOE
LIVING WITH PNH

Julia, diagnosed with aHUS at 15 years old, holding a dog

JULIA
LIVING WITH aHUS

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

DONNAN
LIVING WITH aHUS

Roberta, diagnosed with gMG at 16 years old

ROBERTA
LIVING WITH gMG

Tanner, diagnosed with HPP at 4 days old, with a stuffed animal

TANNER
LIVING WITH HPP

Victor, diagnosed with PNH at 27 years old, with his fiancée and child

VICTOR
LIVING WITH PNH

Erica, diagnosed with aHUS at 22 years old

ERICA
LIVING WITH aHUS

Jill, diagnosed with aHUS at 28 years old, with her daughter

JILL
LIVING WITH aHUS

Justice, diagnosed with aHUS at 9 months old, with his parents

JUSTICE
LIVING WITH aHUS

Adelyn, diagnosed with HPP at 2 days old

ADELYN
LIVING WITH HPP

Evie, diagnosed with HPP at 2 weeks old, playing on a playground

EVIE
LIVING WITH HPP

Maureen & Lauren, diagnosed with LAL-D at 8 and 12 years old, with their parents and brother

MAUREEN & LAUREN
LIVING WITH LAL-D

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

TRENDY FAMILY
LIVING WITH LAL-D

Tristan, diagnosed with LAL-D at 5 years old

TRISTAN
LIVING WITH LAL-D

Bill, diagnosed with PNH at 28 years old, with his wife

BILL
LIVING WITH PNH

Ruthie, diagnosed with PNH at 28 years old, with her husband

RUTHIE
LIVING WITH PNH

ALYSSA
LIVING WITH aHUS

LISA
LIVING WITH PNH

KIM
LIVING WITH NMOSD

MEG
LIVING WITH NMOSD