Rare Disease Archives - Global

A Journey of Strength and Hope, Joris and Maria’s story

Joris is a thriving seven-and-a-half-year-old boy who has been on a journey requiring strength and hope from himself, and all

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

Living with Rare Liver Diseases: Unique challenges, Shared Experiences 

Professor David Jones and Professor Valerie McLin were compensated by Ipsen for their time.   Rare cholestatic liver diseases are conditions

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

. An article by Jennifer Schranz, Senior Vice President and Global Head of Rare Diseases at Ipsen In storytelling, you

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

. An article by Philippe Ghyssels, Vice President Corporate and Global Public Affairs For individuals living with a rare disease, every

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

Imagining More for Rare Diseases: Reflections on the Path Ahead

. An article by Josep Catlla, Executive Vice President, Chief Corporate Affairs Officer “Each year, Rare Disease Day is a

Together for ALGS Awareness Day

. Only 1 in 30,000 children are born with ALGS each year1,2 This January, we are standing with the Alagille

Advocating for babies with Biliary Atresia 

Biliary Atresia (BA) is a rare liver disease that usually appears in the first month of a baby’s life.1 In

Looking after a loved one with Progressive Familial Intrahepatic Cholestasis: Francesca’s Story

A few months after Eva Luna was born, her mother Francesca realized something wasn’t quite right. Within the span of