Sarah's Story

Thanks to research: New hope for Sarah

Sarah is almost four years old. She spent much of her young life in hospital: from birth, the girl suffers from a very rare lung disease, pulmonary alveolar proteinosis. The cause of the disease was not found for a long time, Sarah's suffering could only be treated symptomatically. Regularly, most recently once a month, she had to undergo strenuous lung flushing in hospital in order to breathe properly. In addition, the girl needs permanent oxygen. The prognosis: Without healing therapy Sarah would die within the next two years.

Finding the cause of a rare disease is often like looking for a needle in a haystack. Sarah was very lucky that scientists at Dr. von Hauner Children's Hospital in Munich finally discovered the defective gene: An immunodeficiency is responsible for Sarah's serious illness. With this discovery there is finally new hope - a stem cell transplant could permanently heal the girl!

Children with rare diseases like Sarah are called the "orphans of medicine". All too often, they cannot or only insufficiently be helped because their diseases have not yet been researched and effective therapies are lacking. Their diseases are so rare that the development of new treatment methods is hardly worthwhile for industry.