For the first time in Ukraine, children will be able to receive medicine against spinal muscular atrophy free of charge

Ukraine centrally purchases the medicine Evrysdi ("Rysdyplam") for the treatment of children with spinal muscular atrophy (SMA). Medicines will be provided to children free of charge.

The Ministry of Health reported this on December 9.

The drug will become available for patients with spinal muscular atrophy of the first type in 2023. It will be received by patients with SMA who meet the criteria for inclusion in the program (age, type of disease, etc.), including those who were diagnosed by the results of the expanded neonatal screening program. Detailed conditions for obtaining this drug will be announced later.

"Rysdyplam" is the only medicine against spinal muscular atrophy that can be self-treated at home, because it should be taken orally. There are three medicines in the world that are used to treat SMA: Zolgensma, Evrysdi and Spinraza. The first is administered before the age of 2 and stops the disease forever, the last two must be used throughout life.

In Ukraine, more than 200 children have a confirmed diagnosis of SMA — it is one of the rare genetic diseases characterized by progressive muscle weakness throughout the body. With age, it leads to a violation of the musculoskeletal system and eventually confines the patient to a wheelchair and bed. SMA is the most common genetic cause of death in children. According to statistics, this disease affects one or two babies out of 10 000 newborns.

• In July 2022, Ukraine first received Spinraza, a rare medicine for children suffering from SMA. The drug Spinraza is called the most expensive injection in the world, as one vial costs more than $100 000. Thirteen hospitals have already received more than 150 vials of medicine, donated by donors.