In brief

• Spinal muscular atrophy (SMA), especially type 1, used to be associated with severe disability and a threat to the child's life.
• Three disease-modifying therapies are available, and newborn screening allows treatment to start before symptoms appear.
• Polish doctors and scientists take part in international research and help develop care recommendations for people with SMA.

From a fatal disease to a chronic one

Just over a decade ago, a diagnosis of spinal muscular atrophy, especially type 1, in a child almost always meant serious, progressive disability and a real threat to life (Polscy lekarze współautorami sukcesu terapii na SMA. To szansa dla pacjentów w każdym wieku i z każdym typem choroby – Dziennik.pl) (Polscy lekarze współautorami sukcesu terapii na SMA. To szansa dla pacjentów w każdym wieku i z każdym typem choroby – gazeta.pl) (Polscy lekarze współautorami sukcesu terapii na SMA – tvswietokrzyska.pl). Treatment now offers different possibilities. Three therapies that affect the course of the disease are on the market, and newborn screening allows treatment to begin before the first symptoms appear. According to information from the medical industry, this change in approach is why the condition is increasingly treated as a chronic disease (Polscy lekarze współtworzą przełom w leczeniu SMA - PKB – pkb24.pl).

Polish contribution to research

Polish doctors, scientists and patients take part in international clinical trials, run some of the largest collections of clinical practice data, and help develop recommendations for comprehensive care of patients. Real-world evidence data were presented at the World Muscle Society 2026 congress in Hiroshima. The reports were authored by Dr Anna Lemska and Prof. Sylwia Studzińska.

Therapies for different patient groups

In Poland, therapies are available which, according to representatives of the medical community, cover patients from different age groups. However, it has not been stated which specific preparations are meant or in which indications they are reimbursed, so this question should be checked with the treating physician.