Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial

Shell, Richard D; McGrattan, Katlyn E.; Hurst-Davis, Rebecca; Young, Sally Dunaway; Baranello, Giovanni; Lavrov, Arseniy; O'Brien, Eamonn J.; Wallach, Shiri; LaMarca, Nicole H.; Reyna, Sandra P.; Darras, Basil T.

doi:10.1016/j.nmd.2023.06.005

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Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial

Author(s): Richard D. Shell , Katlyn E. McGrattan , Rebecca Hurst-Davis , Sally Dunaway Young , Giovanni Baranello , Arseniy Lavrov , Eamonn O'Brien , Shiri Wallach , Nicole LaMarca , Sandra P. Reyna , Basil T. Darras

Publication date Created: August 2023

Publication date (Print): August 2023

Journal: Neuromuscular Disorders

Publisher: Elsevier BV

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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

W. Arnold, Louise Rodino‐Klapac, Thomas Prior … (2017)

Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor neuron disease with an onset during infancy that results in failure to achieve motor milestones and in death or the need for mechanical ventilation by 2 years of age. We studied functional replacement of the mutated gene encoding survival motor neuron 1 (SMN1) in this disease.