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Biogen/Ionis’s Spinraza Approved; Second Antisense Drug For Neurodegeneration In 2016

Executive Summary

Analysts expect strong uptake for the antisense oligonucleotide (ASO) given the high unmet need in SMA and positive data in multiple studies, while the first ASO approved this year – Sarepta’s Exondys 51 – has seen some pushback from payers despite the lack of DMD therapies.

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