Skip to Content

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.

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

MAUREEN & LAUREN
LIVING WITH LAL-D

Real Stories of Living With LAL-D

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

Albie

Diagnosed with LAL-D as an infant

When Albie was born, his mom, Charlotte, said that he was, “Absolutely fine. Everything was fine for about 2 weeks.”

READ HIS STORY

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

Maureen & Lauren

Diagnosed with LAL-D at 8 and 12 years old

Lauren was in the first grade when she became ill with persistent abdominal symptoms.

READ THEIR STORY

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

The Trendy Family

Diagnosed with LAL-D

When 2-and-a-half-year-old Brendan was found to have elevated liver enzymes, his parents never imagined it was the first sign that all 4 of their sons were suffering from an ultra-rare disease.

HEAR THEIR STORY

Tristan, diagnosed with LAL-D at 5 years old

Tristan

Diagnosed with LAL-D at 5 years old

Tristan was almost 5 years old when his parents, Holly and Steve, took him to the pediatrician because of his enlarged abdomen and persistent issues with constipation.

READ HIS STORY

Related Topics

Discover how we’re serving people living with LAL-D

LAL-D MEDICINE

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

PIPELINE