A Real-World Study of Nusinersen Effects in Adults with Spinal Muscular Atrophy Type 2 and 3.

Isabelle CôtéVictoria L HodgkinsonMarianne NuryLouis Bastenier-BoutinXavier Rodrigue

Published in: The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques (2024)

This real-world study demonstrates the positive effects of nusinersen in adults with SMA types 2 and 3. Although stabilizing the patient's condition is considered therapeutic success, this study shows an improvement in motor function and subjective gains in several patients.

Keyphrases

ejection fraction
end stage renal disease
newly diagnosed
physical activity
sleep quality
patient reported outcomes