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.

EP: 1.Clinical Features and Phenotypes of Spinal Muscular Atrophy

EP: 2.Natural History of Spinal Muscular Atrophy

EP: 3.Presentation of Adult Spinal Muscular Atrophy in Clinics

EP: 4.Transitions of Care: Moving Spinal Muscular Atrophy Patients from Pediatric/Adolescent Care to Adult Clinics

EP: 5.Key Players in Transitioning Patients with SMA from Pediatric Care to Adult Health Care

EP: 6.Managing Clinical Stability in Adults Spinal Muscular Atrophy Patients

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EP: 7.Treatments Approved for Treatment of Spinal Muscular Atrophy

EP: 8.Factors Guiding Treatment Selection in Adult Spinal Muscular Atrophy

EP: 9.Switching Treatments in Spinal Muscular Atrophy

EP: 10.Determining Treatment Success in Spinal Muscular Atrophy

EP: 11.Counseling Patients About Treatment Outcomes in Spinal Muscular Atrophy

EP: 12.Real-World Data and Stability with Nusinersen in Adult Patients with Spinal Muscular Atrophy

EP: 13.Real-World Data for Risdiplam in Adult Patients with Spinal Muscular Atrophy

EP: 14.Transition of Care in Young Adults With Spinal Muscular Atrophy

EP: 15.Recommendations for Clinicians Taking Care of Patients with SMA

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?