Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps
Spinal muscular atrophy (SMA) is a recessive disorder caused by a mutation in the survival motor neuron 1 gene (SMN1); it affects 1 in 11 000 newborn infants. The most severe and most common form, type 1 SMA, is associated with early mortality in most cases and severe disability in survivors. Nusine...
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| פורמט: | Journal article |
| שפה: | English |
| יצא לאור: |
Wiley
2018
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