Our mission

We were made to give people affected by a rare genetic condition an answer - treatment for their condition.

Watch the video and learn more about rare genetic conditions

Often neglected by large public or industrial investment schemes, and the poor relation when it comes to research and drug treatments - rare genetic conditions are not statistically relevant when considered individually, but the same can't be said about a person's life.

3,5%-5,9% of the world population affected
6,172 known rare conditions
263-446.000.000 people affected in the world

What is our response to this silent emergency?

The journey of scientific research towards a cure is lengthy, demanding, and punctuated by obstacles and successes, involving small steps towards enormous goals.

We work to transform the results of excellent, supported scientific research into drugs and treatments available to patients all over the world

For some years now this vision has been a reality, thanks to innovative treatments that attract the attention of increasing numbers of pharmaceutical groups, and partnerships with public and private health facilities, so our work doesn't just stop at successful scientific results, but actually becomes a reality for each and every patient.

We pursue our vision in two ways:

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