Treatment Options


This is the first officially approved drug for patients diagnosed with SMA. Spinraza increases survival motor neuron (SMN) protein production, addressing an underlying cause of SMA, and promotes the survival of nerve cells. However, it must be consistently given throughout life, in the form of lumbar puncture – by injection into the spinal canal in the lumbar spine, directly into the cerebrospinal fluid. After starting treatment, 4 injections are given over about 2 months, followed by one injection every 4 months.

Jiřík is currently undergoing this treatment. The treatment aims to maintain and slow the progression of the disease and helps some children to improve their condition. Each injection costs about 2 million Czech koruna which is about $80,000 US dollars.


This is a new groundbreaking gene therapy treatment approved by the FDA for children less than 2 years old with spinal muscular atrophy (SMA). At the same time, it is the most expensive drug both in the world and in medical history. Its price reaches almost 54 million Czech koruna- which is about $2.1 million US dollars.

This gene therapy is a one-time treatment consisting of a 60-minute IV infusion that is designed to replace the function of the nonworking or missing gene that causes SMA with a new, working SMN gene to tell motor neuron cells to produce more SMN protein. Motor neuron cells need SMN protein to survive and support muscle functions. However, the drug is not yet registered in the Czech Republic.

Medicinal products that are in clinical trials and therefore not yet approved


A medicine that is given orally and has a similar effect to Spinraza.


A medicine that is given orally and has a similar effect to Spinraza.


Increases muscle activation and thus helps to increase physical endurance and reduce fatigue.


Increases muscle strength and improves motor function in patients with SMA.