This week there has been a huge announcement for the SMA community in Poland: The Minister of Health has declared that since the beginning of a new year the first world registered drug on SMA (Spinraza) will be reimbursed by national health service. We enjoy the happiness of hundreds of families affected by this disease in Poland. Pity only to those babies who did not live to that moment…
And what does it change in our lives? We didn’t wait for reimbursement decision because we realized that every day of delay in drug administration was another thousands / millions of dead motor neurons. In the USA the drug was registered 2 years ago, in the west of Europe over a year. That is why we decided to emigrate and seek treatment in other countries. And this is how Inga found herself in Milan, where she has been taking part in a clinical trial of another medicine on SMA – Risdiplam. It works similar to Spinraza – stimulates copies of the damaged gene to produce enough SMN protein. Protein SMN allows the normal functioning of the motor neurons. Without it neurons die, and without motor neurons muscles die. The treatment is however different by the form of administration: Spinraza is given by the injections in the spine several times a year, while Risdiplam is syrup administered orally every day. Although Risdiplam is in the phase of clinical trials so far, the effects seem to be very promising. Yesterday Roche (the manufacturer of the medicine) announced that the European Medicine Agency granted a “quick path” acceptance for Risdiplam due to the amazing effects during early clinical trials phase. We also think that Inga is getting stronger having this medicine…
There are already questions about whether we are going back to Poland. It’s not and it won’t be an easy decision. Started clinical trial, school, work, home… Three years ago, we’ve already turned our lives upside down. Another “turn over” of our life had happened in May this year. Before we take another decision to move, we’ll consider everything. The most important thing for us is the health of Inga and the happiness of her brother. And that’s what we’re going to be directed by in the upcoming years.
Our Inga entered the period of Autumn-Winter colds. On the attached video she is coughing. In any other circumstances, we would be very worried. However, we are pleased with a strong and “healthy” cough, because one year ago it was so shallow and quiet… We believe that this medicine allows her diaphragm and lungs to keep strength and to deal with the cold.
Please – share, like, invite friends and support. Inga counts on your help!