"It feels like a ticking bomb"
Together we can transform the lives of children like Eva living with a life-threatening rare disease
Progressive familial intrahepatic cholestasis type three (PFIC3) is one of over 7,000 rare diseases that affect millions of people, many of which are children.
PFIC3 is caused by a faulty gene that leads to progressive liver damage which causes children to suffer from jaundice and severe itching. It can also impact the wider body and affect a child's brain development, eyesight and growth.
A liver transplant is the only effective treatment, but it has a risk of complications – and there is a shortage of donor livers.
New treatments are desperately needed. That's why we're supporting research that offers hope of a safer, less invasive treatment for children with PFIC3, but we need your help to support the final stage of this vital work. Will you give a gift today and help transform lives?
With your help, we will NEVER stop fighting rare diseases
Eva's story
At just nine months old, Eva was diagnosed with PFIC3 after spending most of her first few months of life in and out of hospital struggling to eat and sleep.
For the last two years Eva's health has been okay, but with the disease being so rare, and with few treatments available, her parents know there will come a time when her health will worsen and a liver transplant will be the only treatment that could save her life.
It feels a bit like a ticking time bomb. That’s why the research Action is funding is hugely important. There are so few non-invasive treatment options – really, it’s just the medication she’s on or a liver transplant.
Targeting the cause of PFIC3
Learn how researchers are developing a new gene therapy to treat this serious condition
A gift today could help transform the lives of children
100% of your donation will support research that offers hope of a new, less invasive treatment option that is desperately needed for children with PFIC3.