Thanks to caring people from all over the world we saved Dmytro Svichynskyi by raising $ 2,050,000 on the drug Solgensma for 80 days. However, even according to unofficial data in Ukraine, more than 200 families still need treatment for spinal muscular atrophy.
Parents of sick children with SMA continue to make every possible and impossible effort, but without the help of the state, seriously ill children are simply doomed. They have a few months, because without proper treatment, children with SMA and other orphan diseases die before they are two years old.
Together we can achieve extraordinary results as we have proved with the example of a charity fundraiser for the treatment of Odessa resident Dmytro Svichynsky!
We offer the following solutions:
Purchase medicines registered in Ukraine – Rysdiplam or Spinraza for 90 children 1-2 types of SMA, in 2021, by concluding controlled access agreements or otherwise. Approximate cost of UAH 400 million.
Develop a national protocol for the treatment of children with AMD (currently non-existent).
Create an electronic register of patients with SMA.
Develop a comprehensive long-term program to support children with ASD, which will provide funding for:
- purchase of medicines for people of all types of SMA;
- introduction of early neonatal screening of children for AMS in maternity hospitals; UAH 150 million
- creation of reference centers, on a territorial basis, for children with rare orphan diseases, where they will work with multidisciplinary teams of doctors.
Introduce mandatory family information about possible genetic risks that could lead to the birth of a sick child.
Negotiate with Roche to continue the Risdiplam early access program, which ended in July 2021.
Currently, 58 families in Ukraine receive free treatment under this program.
Negotiate with Novartis, the developer of the Zolgensma gene therapy drug, on registration of the drug in Ukraine; purchase of Solgensma at the expense of the state budget.