Our chronicle
MN190 is a test name of a substance in the enzymatic treatment of an experimental US pharmaceutical company BioMarin. The therapy has been implemented since 2013/14 year. It stops the disease or at least significantly slows it down. This is the opinion of the company itself and the parents of children who have been participating in a clinical trial (24 children in 4 clinical centers: Hamburg, Rome, London, Ohio).
See: http://www.biomarin.com/products/clinical-trials/cerliponase-alfa/
The great thing. The great hope. The great drama.
Although the therapy exists the diagnosed children can not participate in the clinical trial after its closure. They will not live long enough to get the registered medication. They have been dying while they could have received help. We are fighting along with other parents to get the faster access to the “medicine” through, so called “compassionate use” formula. It allows for the usage of a medicine which is not yet registered if it is the only possibility for the patient to survive. Nevertheless, the consent for the "drug compassion" always comes from the manufacturer.
See: http://www.biomarin.com/products/compassionate-use-policy/
If we could wait, we would wait. The disease is however faster than the procedures. It has been taking everything from us during months. We have been hoping for the treatment extension in the 4th quarter of the year since April to September (2015). Such were the official information provided by the medicine manufacturer and clinical centers.
Our hope has been taken away in October – the company’s plans have been changed. Now they are considering the extension of treatment in the middle of 2016 claiming that is not proved to be safe yet. Our safety ends now anyway. We are very understanding but we can not stand the prioritizing of postponing procedures over life, especially when it disappears so quickly. This is the greatest tragedy for those who have not lived enough. We have nothing left. There is not a slightest chance that the disease will wait.
This is the CHRONICLE of our JOINT efforts for Kuba’s medicine…
For the possibility of treatment for those, who will not wait!

biomarin„ Just wait till 2nd of March, it is not even a full month.”
We heard that sentence raising our hope once again in Paris. During the global symposium on rare diseases, lasting several days with a symbolic inauguration on 29th of February in San Diego, the consortium claimed on the 2nd of March that they managed to restrain the cruel Batten Disease. The therapy stopped the development of the disease for many children, for the rest of them visibly slowed it down. It is a great success and huge hope for patients, so the company begins the procedure of registering the medicine in order of its availability in the middle of 2017. Simultaneously it starts for an “early access”, so SOME of the children would be able to have the medicine ALREADY in the 3rd quarter of 2016. It does not give the information about the criteria for selection. After that statement, the company's quotes on the stock are improving. On the picture you can see the President and Chief Executive Officer of the BioMarin Pharmaceutical Inc. - Jean-Jacque Bienaimé.

Our long discussions from Paris were not included in that statement and yet the drug can be given as a compassionate use. It could have been given for many months now. A good will of the company would be enough, a good will which is never there. One of the articles eloquently describes the “children on a turning way”. I do not know if my little Kuba is still ON a turning way or AFTER it. I do not know for how long we can survive this mental torture… Maybe if they could swap with us for a week, they would be able to see the situation from the different perspective…

'Kids on the cusp': Early data moves BioMarin against a child killer
BioMarin comunicate

BioMarin’s statement:
Good BioMarin:

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