Stories

Every year, Rare Disease Day reminds us that rare conditions impact far more lives than many realize -over 300 million people globally, across more than 10,000 identified rare diseases. Yet what strikes me most on this day of recognition and reflection is that listening to patients remains an under-utilized lever for achieving progress in healthcare.

Rare diseases are more common than most people imagine, in how many lives they touch and in how profoundly they shape everyday life for patients and families. Behind every diagnosis is a person navigating far more than symptoms: daily routines transformed, ambitions redefined, misunderstanding and isolation to navigate and families adapting to uncertainty with resilience and hope.

“We were shocked” That is how Celine felt when her family received the news that her daughter Suzanne had been diagnosed with the rare liver disease, Alagille Syndrome (ALGS) at just a few months old.

Ever wonder how our science works? Watch Ipsen scientists share how they turn ideas into impact.

Ever wonder how our science works? Watch Ipsen scientists share how they turn ideas into impact.