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Stories & Case Studies

CHARLIE

(case study to add)
Australia

CHARLI Q

Charlie’s story is a powerful reminder of the urgent need to develop better treatments. Join us in our mission to make a difference to children like Charlie.
Favourite food
Pizza (with lots of ketchup)
Favourite film
Hercules

MARK’s STORY

Our family is from Ukraine. Our son Mark was born in December


Where we need you

Every donation helps us create a future where rare genetic disorders are understood and fully treatable. 

Every gift of time supports us along our pathway to a cure.

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