Vol 34, No 62 (2024): Continuous Publishing
Editorial
Published online: 2024-08-12
Ensuring continuity and development of spinal muscular atrophy treatment in Poland
1
, 2, 3, 4, 5, 167, 8, 9
, 2, 3, 4, 5, 167, 8, 9
DOI: 10.5603/cn.101979
Neurol Dziec 2024;34(62):1-4.
Abstract
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.
Keywords: spinal muscular atrophySMAonasemnogene abeparvovecgene therapy
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