“Around one in 20 newborns is born with a rare disease. We doctors are then left “empty-handed” and can only alleviate the symptoms, if at all. Many of these children will never be able to lead an independent life. Even after 40 years in the profession, I am still deeply moved by the suffering of the families affected.

What gives me hope is the enormous gain in knowledge, this real turning point in medicine that we are currently experiencing: it provides us with precise methods and procedures that enable early diagnosis and the development of highly effective causal therapies.

This is a historic opportunity that gives us a responsibility to act swiftly and decisively. So that everyone can benefit from medical progress.”

Alliance4Rare – Research initiatives for children with rare diseases

Alliance4Rare combines the expertise of research-oriented university children’s hospitals with the creative drive of private and institutional sponsors. Together, the partners want to enable medical progress where it is most urgently needed: Children affected by particularly serious rare diseases need pediatricians and human geneticists who are strong in research and conduct targeted research on the most urgent issues.

Eva Luise Köhler Research Award for Rare Diseases

Research into rare diseases often faces major institutional hurdles – but is rarely in the spotlight. With the Eva Luise Köhler Research Prize, we are creating publicity for this important topic and driving forward the development of the research field. As start-up funding, the prize money of €50,000 enables research into disease mechanisms and novel therapeutic approaches for rare diseases.

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National Register for Rare Diseases (NARSE)

Many rare diseases are virtually unexplored. Together with our think tank partners, we are initiating the implementation of a sustainable patient registry for rare diseases. This will give us access to structured data on possible disease manifestations and progression and create the basic prerequisites for targeted research and evidence-based medical care.

TO NARSE