Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps

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...

書誌詳細
主要な著者:	Gidaro, T, Servais, L
フォーマット:	Journal article
言語:	English
出版事項:	Wiley 2018

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