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Today, researchers are increasingly succeeding in deciphering the pathological mechanisms of disease development. They are thus providing starting points for the development of targeted drugs and antibodies that can significantly reduce the symptoms of affected people.
Encouraging treatment successes with gene and cell therapy methods herald a real turning point in medicine. In particular, those affected by rare diseases with a monogenetic cause may hope for a cure if promising research approaches are now consistently pursued.
To advance the development of promising treatment approaches, Alliance4Rare initiated by our foundation, bundles the expertise of research-strong university children's hospitals along a cross-site research strategy for rare diseases.