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Nusinersen in Infantile-onset Spinal Muscular Atrophy: Results from Longer-term Treatment from the Open-label SHINE Extension Study (1640)

Diana Castro, Richard S. Finkel, Michelle A. Farrar, M. Tulinius, Kristin J. Krosschell, Kayoko Saito, Giulia Gambino, Richard Foster, Ishir Bhan, Janice Wong, Boris Kandinov, Wildon Farwell

2020Neurology20 citationsDOI

Abstract

To present interim results from the SHINE open-label extension study (NCT02594124) for participants with infantile-onset SMA who transitioned from previous nusinersen trials.

Topics & Concepts

Spinal muscular atrophyMedicineAtrophyOpen labelPediatricsSurgeryPhysical medicine and rehabilitationInternal medicineRandomized controlled trialDiseaseNeurogenetic and Muscular Disorders ResearchRNA modifications and cancerMuscle Physiology and Disorders
Nusinersen in Infantile-onset Spinal Muscular Atrophy: Results from Longer-term Treatment from the Open-label SHINE Extension Study (1640) | Litcius