Patients

Céline and Suzanne: Navigating Alagille Syndrome

At just nine years old, Suzanne is a bright, creative young girl who loves dancing, drawing, exploring the woods, and watching birds. Despite living with Alagille syndrome (ALGS), a rare genetic condition, she embraces life with enthusiasm and curiosity.

Living with Rare Liver Diseases: Unique challenges, Shared Experiences 

Cholestatic liver diseases are a rare but significant group of conditions that impact both children and adults. Three such conditions—Primary Biliary Cholangitis (PBC), Progressive Familial Intrahepatic Cholestasis (PFIC), and Alagille Syndrome (ALGS)—share commonalities yet are unique in their caus

Scientific Journey from Coal to Diamond: Myths and Misperceptions in Rare Disease Innovation 

In storytelling, you might hear of a superhero who squeezes coal with immense strength and heat, instantly creating a flawless diamond. While fiction, it’s true that diamonds are rare and formed under precise conditions over millions of years. For me, this analogy resonates deeply with the developme

Breaking Barriers to Rare Disease Innovation: Reflections from the World EPA Congress 

For individuals living with a rare disease, every day without treatment is a day too long. At the World Evidence, Pricing and Access (WEPA) congress in Amsterdam, I participated in a panel discussion on long-term access to rare disease innovation. The conversation reminded me that behind every polic

Rare Disease Day 2025: Imagining More for the Rare Disease Community 

Every day, millions of people around the world face the immense challenges of living with a rare disease. For these individuals and their families, resilience is essential, and hope becomes a lifeline. Rare Disease Day is a moment to honour their courage, amplify their voices, and reaffirm our share

Imagining More for Rare Diseases: Reflections on the Path Ahead

An article by Josep Catlla, Executive Vice President, Chief Corporate Affairs Officer

Driving continuous progress in cancer care

While being diagnosed with cancer isn’t unique- around 20m people are diagnosed with cancer globally each year, and that number is projected to double in next 20 years—how it affects each person is.

Neuro Connections

Watch our three-part vodcast series, Neuro Connections, developed in collaboration with the World Stroke Organization, where we discuss current post-stroke care challenges and potential solutions with global specialists, advocates and healthcare professionals.

Together for ALGS Awareness Day

Only 1 in 30,000 children are born with ALGS each year. This January, we are standing with the Alagille Syndrome Alliance to help raise awareness of this rare cholestatic liver disease and be #ALGSAware.

Assessing the benefits of early intervention in post-stroke care 

With over 12 million people a year experiencing a stroke globally and over 100 million living with the effects and consequences of stroke, the burden of stroke is high and set to increase.