Massive SMA study reveals Real-World treatment patterns

NCT ID NCT07403214

First seen Feb 24, 2026 · Last updated May 15, 2026 · Updated 9 times

Summary

This study looked at medical records of over 4,800 people with spinal muscular atrophy (SMA) to see how three approved treatments (onasemnogene abeparvovec, nusinersen, and risdiplam) are used in real life. Researchers tracked complications, medical equipment needs, and hospital visits before and after starting these treatments. The goal was to understand treatment patterns and outcomes outside of controlled clinical trials.

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Contacts and locations

Locations

  • Novartis

    East Hanover, New Jersey, 07936, United States

Conditions

Explore the condition pages connected to this study.