Opinion|Videos|July 1, 2024

Treatments Approved for Treatment of Spinal Muscular Atrophy

Author(s)Lauren Elman, MD
Arun S. Varadhachary, MD, PhD

Key opinion leaders (KOLs) examine the mechanisms of action and other pertinent information regarding FDA-approved treatments for spinal muscular atrophy (SMA), sharing their perspectives on these therapies with a particular focus on the various modes of administration.

Please review mechanism of action and other details for treatments that are FDA approved for SMA.
1. Nusinersen, Onasemnogene abeparvovec, Risdiplam
Based on the pathophysiology of SMA, do you expect mechanism of action to impact disease pathology differently?

How do you feel about mode of administration?

How do you discuss the progressive nature of SMA with your adult patients?

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