Dezember 2023 —
Our latest film about the genome research funded by the Care-for-Rare Foundation aims to give children with rare diseases around the world hope for a cure.
For this film, we reunited with Sarah, one of the many children that Care-for-Rare and Dr. von Hauner Children’s Hospital have been able to help. What a happy girl Sarah has become!
She suffered from an extremely rare disease that led to life-threatening lung problems.
She was the first patient in Europe to receive a bone marrow transplant to treat this disease. She responded very well to this and her body was able to produce functional immune cells.
Four years after her successful treatment, she was now back in Munich for a routine check-up.
Sarah is one of around four million people in Germany who suffer from a rare disease, most of whom are children. Medical care for these children is usually complex, expensive and requires a multi-professional team.
The search for the genetic causes of rare diseases often resembles detective work. However, genome research is the key to new approaches and the basis for modern precision diagnostics and thus for the development of new individualized treatment options.
Sarah is one of the winners of this genome research.