• Center on Health Equity & Access
  • Clinical
  • Health Care Cost
  • Health Care Delivery
  • Insurance
  • Policy
  • Technology
  • Value-Based Care

Introduction to Therapies Used to Treat SMA

Opinion
Video

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers (nusinersen [Spinraza] and risdiplam [Evrysdi]) for older patients.

Clinical Brief: Historical and Current Treatment Paradigms for SMA

Main Discussion Topics

  • Evolution of SMA care from supportive/palliative to disease-modifying approaches
  • Description of current FDA-approved disease-modifying therapies
  • Mechanisms of action for available SMA treatments
  • Challenges in accurately measuring treatment response

Key Points for Physicians

  • Before disease-modifying therapies, SMA care focused on respiratory support, nutrition, orthopedic management, and pain control.
  • Current treatment options include nusinersen (intrathecal antisense oligonucleotide), risdiplam (oral splicing modifier), and onasemnogene abeparvovec (gene therapy, limited to children younger than 2 years).
  • Nusinersen and risdiplam enhance SMN protein production from the SMN2 gene.
  • Standardized assessment tools have limitations at both ends of the functional spectrum.

Notable Insights

Implementation of consensus care guidelines and multidisciplinary care improved survival outcomes even before disease-modifying therapies were available, highlighting the importance of comprehensive care approaches.

Clinical Significance

The treatment paradigm for SMA has evolved from palliative care to a multifaceted approach combining disease-modifying therapies with supportive care, requiring ongoing adaptation of assessment methods to capture meaningful clinical outcomes.

Related Videos
5 experts in this video
4 experts are featured in this series.
5 experts in this video
4 experts are featured in this series.
Dr CK Wang
1 expert is featured in this series.
Eileen Peng, PharmD, sitting for a video interview
2 experts in this video
Related Content
© 2025 MJH Life Sciences
AJMC®
All rights reserved.