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
Liz is our inspiration

Although physical challenges required Liz, her husband, and their children to adjust their lives in many ways, she doesn't let her disease hold her back.
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.

KENDALL
LIVING WITH NF1

REAL STORIES

Real Stories of Living With NF1

Kendall

Diagnosed with NF1 at 10 weeks old

When Kendall was born, her mom, Stephanie, knew something was different about her. “Being a nurse, everyone was just telling me I was being overly concerned,” Stephanie says. “But there were just little details that I noticed as a mom, as any mom would, that just didn’t seem right.”

HEAR HER STORY

Discover how we’re serving people living with NF1

About NF1

See how we’re pushing the boundaries of NF1 research every day

PIPELINE

Real Stories of Living With NF1

Kendall

Diagnosed with NF1 at 10 weeks old

Discover how we’re serving people living with NF1

See how we’re pushing the boundaries of NF1 research every day

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