Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.

By Richard S. Finkel, C. Chiriboga, J. Vajsar, J. Day, J. Montes, D. D. De Vivo, Mason Yamashita, F. Rigo, G. Hung, E. Schneider and 4 more. The Lancet, 2016

887 citations recorded by the source corpus.

Subfields: Genetics, Neurology

View Paper: https://www.semanticscholar.org/paper/9a97722a092538056ffd6fb53eb4f3ce97b7a50b

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The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

1. M. Main
2. E. Mazzone
3. M. Vitale
4. B. Snyder
5. S. Quijano-roy
6. E. Bertini
7. Rebecca Hurst Davis
8. Oscar H Meyer

Citations in the corpus

• Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease (2019, result): “A similar plateauing of CSF trough levels was observed in a previous study of intrathecally delivered nusinersen (9 to 11 ng per milliliter after four repeated doses of 12 mg).(8) Panel C shows the trends in seizure frequency and duration as reported in a seizure diary recorded by the parents.”
• Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. (2017, positive): “The advent of new therapies leading to increased survival and overall functional improvements [7,8], is rapidly changing the scenario of spinal management in these patients.”
• Nusinersen versus Sham Control in Infantile‐Onset Spinal Muscular Atrophy (2017, neutral): “Decreased oxygen saturation 10 (12) 10 (24)”