What is SMA?
Spinal muscular atrophy (SMA) is a rare genetic condition that makes the muscles weaker and causes problems with movement. Type 1 is the most severe form and symptoms develop very early and cause death usually before the baby reaches two. Although the progression of the disease can be slowed with Spinraza (a drug offered by the NHS), the only cure that's available is gene therapy, which is sold under the name of 'Zolgensma' by Novartis.
The ultimate effect of Zolgensma will be to replace the mutated SMN1 gene with a fully functional one. SMA children's marrow and muscle cells will naturally start producing the essential protein SMN.
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