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Monograph- Selecting disease-modifying medications in 5q spinal muscular atrophy
Monograph: Selecting disease-modifying medications in 5q spinal muscular atrophy
CME Available -
Spinal muscular atrophy (SMA) is an inherited lower motor neuron disease. SMA occurs secondary to alterations in the survival motor neuron 1 gene (SMN1), which is the main driver of SMN protein production. The severity of the disease is determined by the number of copies of the SMN2 gene, which is a homolog to SMN1 but not as efficient in protein production. Three medications have recently been approved for the treatment of SMA. Nusinersen is an intrathecal antisense oligonucleotide that alters SMN2 pre-mRNA, onasemnogene abeparvovec-xioi is an intravenous SMN1 gene replacement therapy, and risdiplam is an oral small molecule splicing modifier of SMN2. No head-to-head studies have been conducted comparing these medications, so selection of one of these medications for an individual with SMA can be challeng-ing. In this article we outline the efficacy, safety, and other pertinent factors to con-sider when selecting a therapy for an individual with SMA. The age of the individual and comorbidities, such as liver or kidney disease, help guide treatment choices. All three of these medications are efficacious, and early initiation is critical for obtaining the best outcomes.

1) describe the three treatments available for SMA.
2) discuss the mechanism of action of each treatment.
3) describe the considerations that are involved in choosing a specific treatment.

The AANEM is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

All conflicts of interest have been resolved according to ACCME standards. All authors/planners of this activity had nothing to disclose. 

The AANEM designates this enduring material for a maximum of 2.0 AMA PRA Category 1 Credits TM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Credit expires 10/6/2024.
Michael S. Cartwright MD, MS and Suraj Upadhya
Availability: On-Demand
Expires on Oct 06, 2024
Cost: Member: $0.00
Non-Member: $15.00
Credit Offered:
2 CME Credits
2 CEU Credits
2621 Superior Drive NW Rochester, MN 55901
Phone: 507.288.0100
Fax: 507.288.1225
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