The most prominent approval in the second quarter of 2019 was Novartis’s pioneering gene therapy for spinal muscular atrophy (SMA), Zolgensma (onasemnogene abeparvovec), a rare neuromuscular disease with a typical life expectancy of less than 2 years if untreated.

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Fonte:NATure Reviews | Drug DIScovery volume 18 | August 2019 | 575