Liora Sagi

6 Weizmann St, 64239-06
Tel Aviv, TA 64239, Israel

Last Updated: 09/26/2026

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Found 20 publications

Respiratory outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy: national real-world cohort study.

Respiratory outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy: national real-world cohort study.

Journal: European journal of pediatrics

Published: August 08, 2024

Treating late-onset Tay Sachs disease: Brain delivery with a dual trojan horse protein.

Treating late-onset Tay Sachs disease: Brain delivery with a dual trojan horse protein.

Journal: Molecular therapy. Methods & clinical development

Published: April 26, 2024

Non-ionizing measurement and quantification of bell-shaped chests in spinal muscular atrophy: a pilot study.

Non-ionizing measurement and quantification of bell-shaped chests in spinal muscular atrophy: a pilot study.

Journal: Frontiers in pediatrics

Published: July 10, 2023

Glucagon-like peptide-1 analog therapy in rare genetic diseases: monogenic obesity, monogenic diabetes, and spinal muscular atrophy.

Glucagon-like peptide-1 analog therapy in rare genetic diseases: monogenic obesity, monogenic diabetes, and spinal muscular atrophy.

Journal: Acta diabetologica

Published: November 28, 2022

Real-Life Outcome After Gene Replacement Therapy for Spinal Muscular Atrophy: A Multicenter Experience.

Real-Life Outcome After Gene Replacement Therapy for Spinal Muscular Atrophy: A Multicenter Experience.

Journal: Pediatric neurology

Published: August 01, 2022

Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients.

Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients.

Journal: European journal of neurology

Published: February 11, 2022

Cerebrospinal fluid characteristics of patients treated with intrathecal nusinersen for spinal muscular atrophy.

Cerebrospinal fluid characteristics of patients treated with intrathecal nusinersen for spinal muscular atrophy.

Journal: Muscle & nerve

Published: January 20, 2022

Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec -routine care evidence.

Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec -routine care evidence.

Journal: Gene therapy

Published: November 08, 2021

Nonrespiratory complications of nusinersen-treated spinal muscular atrophy type 1 patients.

Nonrespiratory complications of nusinersen-treated spinal muscular atrophy type 1 patients.

Journal: Pediatric pulmonology

Published: September 02, 2021

Diagnostic yield of chromosomal microarray and trio whole exome sequencing in cryptogenic cerebral palsy.

Diagnostic yield of chromosomal microarray and trio whole exome sequencing in cryptogenic cerebral palsy.

Journal: Journal of medical genetics

Published: April 04, 2021

Variable Genotype-Phenotype Correlation of Pompe's Disease Caused by a c.2015 G > A (p.Arg672Gln) Mutation in the GAA Gene.

Variable Genotype-Phenotype Correlation of Pompe's Disease Caused by a c.2015 G > A (p.Arg672Gln) Mutation in the GAA Gene.

Journal: Neuropediatrics

Published: February 12, 2021

Nutritional Therapy in Children With Spinal Muscular Atrophy in the Era of Nusinersen.

Nutritional Therapy in Children With Spinal Muscular Atrophy in the Era of Nusinersen.

Journal: Journal of pediatric gastroenterology and nutrition

Published: January 25, 2021

Showing 1-12 of 20

Last Updated: 09/26/2026