Stories

Hugo Gomes da Silva, SVP Global Medical Affairs (Rare Disease) at Ipsen, shares powerful insights into the evolving challenges families face throughout their PFIC journey—and highlights the critical importance of trusted information and community support.  

Nathan McCormack focuses on the earliest stages of active pharmaceutical ingredient (API) development.

Naomi Binoche, VP Head of Strategic Alliance Management, shares insights on how pharma partnerships are key to driving success and why celebrating every milestone along the way matters.

For the thousands of individuals, mostly women, living with primary biliary cholangitis (PBC), a chronic, progressive autoimmune liver disease, the journey can be lonely.

Lily May, a vibrant and determined seven-year-old, loves gymnastics and golf. Yet, like many children living with pediatric low-grade glioma (pLGG), her life has been profoundly shaped by this rare brain tumor. Although the global burden is unknown, pLGG is a rare childhood brain tumor.