Thanks to your support, we have hope of finding a cure for our children with the serious genetic disease SPATA5.

WHO ARE WE?

We are an association of parents of children with the serious genetic disease SPATA5. There is currently no cure for it. We refused to accept this and decided to fight for a change.

About the disease SPATA5

More information about us

Our children’s stories

OUR GOAL

Our goal is to find a cure for our children. We have approached scientists from the Academy of Sciences of the Czech Republic, who have already started research. Unfortunately, there is no budget for research and development of cures for rare diseases in the Czech Republic. We have to pay for everything from our private sources. The current estimate of the cost of research and development of the cure is between
1,3 - 1,9 million EUR.

1. Pilot phase

During the pilot phase, detailed research into the gene and design of a treatment procedure will take place.

We estimate the duration of this phase to be 12-18 months.
The estimated cost of this phase is 120.000 - 160.000 EUR.

2. Preclinical study

The result of the preclinical study will be approval for human testing of the drug from the Czech Institute for Drug Control and the European Medicines Agency (EMA).

This phase will last 2-3 years.
The estimated cost is 800.000 – 1.000.000 EUR.

3. Clinical study

As part of the clinical trial, the drug will be administered to the first patients.

This phase will last at least 4-5 years.
It is difficult to estimate the costs of this phase, but they may be around 400.000 – 800.000 EUR.

More about drug research

HOW YOU CAN HELP OUR CHILDREN

Donate to our children's fundraising campaign for research and development of a cure for SPATA5.

We have started this fundraising campaign in order to finance the research being carried out by Czech scientists from the Academy of Sciences of the Czech Republic. We have approached these scientists to help our children. We, the parents, are paying for all the research from our own funds, because there is no budget for research into rare genetic diseases in the Czech Republic.