Stories

People with rare genetic diseases may perhaps not display the symptoms immediately. During the early years of life, everything seems perfectly normal. Parents then notice something odd about their child. This is when the examinations begin, journeys undertaken in search of an answer, a diagnosis, hope. Watch the videos and read the stories about the people and families we dedicate our efforts to helping.

LORENZO’S FUTURE

“Mum, will I walk?” We want the answers, for children like Lorenzo.

THERE’S STILL NO CURE FOR ANDREA’S DISEASE

But neither he nor his family will give up!

EMMA’S DREAMS

To grow up. To study. Having ambitions and making them come true. We are at her side.

THE WORDS CARLOTTA CAN’T SAY

But in her eyes we see her inner strength. Why Carlotta isn’t giving up.

FOR THEM, EVERY DAY IS A VICTORY IN ITSELF

The gene therapy that we’ve developed in our laboratories provides concrete hope.