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: admin Source Type: research
More News: Adenoviruses | Brain | Drugs & Pharmacology | Gene Therapy | Genetics | Motor Neurone Disease | Neurology | Spinal Muscular Atrophy