Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial-Reference-Cited by-同舟云学术

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

Published:2023-08 Issue:8 Volume:33 Page:670-676
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

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

Funder

Novartis Gene Therapies

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

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5. Correlation between SMA type and SMN2 copy number revisited: an analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases;Calucho;Neuromuscul Disord,2018

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