In Brief: Risdiplam (Evrysdi) for Spinal Muscular Atrophy (online only)

Date: July 26, 2021 Issue #:  1629Summary:  Risdiplam (Evrysdi– Genentech), a survival of motor neuron 2 (SMN2) splicing modifier, has been approved by the FDA for oral treatment of spinal muscular atrophy (SMA) in patients ≥2 months old. It is the first oral drug to be approved in the US for treatment of SMA; nusinersen(Spinraza), an intrathecally administered SMN2-directed antisense oligonucleotide, and onasemnogene abeparvovec(Zolgensma), an IV adeno-associated virus vector-based gene therapy, were approved earlier.
Source: The Medical Letter - Category: Drugs & Pharmacology Authors: Source Type: research