Ensuring continuity and development of spinal muscular atrophy treatment in Poland

Spinal muscular atrophy is a neuromuscular disease genetically caused by damage to the SMN1 gene. The disease is characterised by a progressive loss of motor neurons leading to muscle weakness and consequent physical disability and respiratory failure. In Poland, we treat the widest possible population of SMA patients with all drugs registered for this indication, despite the very high unit costs. The development of the SMA treatment programme has become a model for diagnostic and therapeutic management, which can also be applied to the treatment of other rare diseases.