This week Inga spends at rehabilitation camp at the Neurorehabilitation Centre. She does many exercises to become a bit stronger and a little more efficient. We are very proud of her!

We cannot stop thinking about the last week’s information about the new drug (Zolgensma), which has just been approved for use in the US. We return our thoughts to the day of Inga’s diagnosis and to the situation we had then in the treatment of spinal muscular dystrophy. In February 2016 there were no medicines available, Spinaraza clinical trials were held only in selected countries and the recruitment was completed, the start of new clinical trials was a great unknown, the doctors’ awareness of the possibility of participating in such studies was very low. The diagnosis of SMA continued to sound like a death sentence…

Today, Spinraza treatment is widely available (hope to include UK very soon). In the US (and probably soon in Europe), treatment with an ultra-modern drug replacing a damaged gene for children up to the age of 2 (Zolgensma) will be available. We also have a medicine which is taken by Inga (Risdiplam) and which performs very well compared to the other two (attached table and link below). The above situation on the drug market just three years ago seemed simply unrealistic.

All this translates into our optimism and hope that the first Inga’s step we desire is not just a dream but the realistic goal of our lives.

Thank you all for your support! Those who would like to support treatment in Italy can visit the following websites:
https://www.justgiving.com/crowdfunding/milan3
https://zrzutka.pl/9p5w48
Thank you!

https://www.evaluate.com/vantage/articles/news/trial-results/aan-2019-roche-confirms-its-place-spinal-muscular-atrophy?fbclid=IwAR1Pq6kerDpFpQgreUnC45sfCc587VNkvjp4plRXwsquePkVToEPsnXCvFA