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

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

doi:10.1016/j.nmd.2023.06.005

Public

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

Published in Neuromuscular Disorders • Jun 22, 2023

Authors:

Richard D. Shell

Katlyn McGrattan

Rebecca Hurst-Davis

Abstract

Bulbar function in spinal muscular atrophy has been defined as the ability to meet nutritional needs by mouth while maintaining airway protection and communicate verbally. The effects of disease-modifying treatment on bulbar function are not clear. A multidisciplinary team conducted post-hoc analyse...

Subject

Medicine

Spinal muscular atrophy

Swallowing

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!