Statistically significant improvement in motor functions were detected in the patients treated with continuous regimen after 24 weeks of treatment, and while the differences were not significant at 48 weeks, a positive trend in favour of Drisapersen was observed at this time-point [ 81 ].
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The sentences
Very recently, Spinraza (previously known as Nusinersen), an O -(2-methoxyethyl) modified ASO administered intrathecally, has moved from phase I, to phase II and III studies for infants and children with Spinal Muscular Atrophy (SMA), with very encouraging tolerability and highly significant clinical benefit for treated patients [ 7 , 8 ].