Concepedia

Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies

Marika Pane, Beatrice Berti, Anna Capasso, Giorgia Coratti, Antonio Varone, Adele D’Amico, Sonia Messina, Riccardo Masson, Valeria Sansone, Maria Alice Donati,

EClinicalMedicine · 2023 · 39 citations · 26 references

DOIFull text

Open access

Abstract

None.

References

26

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.2K citations · Full text

Nelson Textbook of Pediatrics

Archives of Disease in Childhood · 1965 · 825 citations · Full text

, , +11

Observational study of spinal muscular atrophy type I and implications for clinical trials

Richard S. Finkel, Michael McDermott, Petra Kaufmann et al. · Neurology · 2014 · 506 citations · Full text

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

John Day, Richard S. Finkel, Claudia A. Chiriboga et al. · The Lancet Neurology · 2021 · 449 citations

, , +5

Natural history of infantile‐onset spinal muscular atrophy

Stephen J. Kolb, Christopher S. Coffey, Jon Yankey et al. · Annals of Neurology · 2017 · 392 citations · Full text