Primary biliary cholangitis (PBC) is a chronic autoimmune disease that affects liver function and can have debilitating symptoms for people
Category: Rare Disease
Learning the impacts of FOP from patients and their families – The Burden of Illness study
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder where extra bone formation takes place in soft tissues, including muscles,
Primary Biliary Cholangitis (PBC): Looking Beyond the Liver
“Symptom burden is the main issue for the patient.”– Professor Andreas Kremer, Hepatologist of the University of Zurich Hospital Primary
Advancing Primary Biliary Cholangitis (PBC) Management: Insights from Experts
“We are on the cusp of an absolute explosion in our approach to managing PBC.” – Professor Gideon Hirschfield, Hepatologist
Collette: finding community through PBC
Collette spent almost 20 years going back and forth between different doctors and treatments, before she was finally diagnosed with
Fireside Chat: Innovation and Policy Change in Rare Diseases in Europe
The rare disease community continues to strive for innovative solutions to meet patient needs and ensure no person living with
Understanding the true impact of living with FOP
This year marks the 17th anniversary since the discovery of the ACVR1 gene was announced, a major scientific milestone in fibrodysplasia ossificans progressiva
Living with PBC: The Person Behind the Chart
‘Invisible’ diseases like primary biliary cholangitis (PBC) can be difficult to recognise, diagnose and to manage effectively. Here Prof. Gideon
A Life in a Day with FOP: from Empathy to Understanding
You can never truly understand what it’s like to live with an ultra-rare condition unless you’ve experienced it for yourself.
‘A Life in a Day’: the realities of living with FOP
I was honored to be invited to take on the challenge to live ‘A Life in a Day of a