Real-Life Outcome After Gene Replacement Therapy for Spinal Muscular Atrophy: A Multicenter Experience-Reference-Cited by-同舟云学术

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

Published:2023-07 Issue: Volume:144 Page:60-68
ISSN:0887-8994
Container-title:Pediatric Neurology
language:en
Short-container-title:Pediatric Neurology

Author:

Tokatly Latzer Itay^ORCID,Sagi Liora,Lavi Revital,Aharoni Sharon^ORCID,Bistritzer Jacob,Noyman Iris,Ginsburg Mira,Lev-Or Angela,Katzenellenbogen Sharona,Nevo Yoram^ORCID,Fattal-Valevski Aviva

Publisher

Elsevier BV

Subject

Neurology (clinical),Developmental Neuroscience,Neurology,Pediatrics, Perinatology and Child Health

Reference44 articles.

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2. Impact of a national population-based carrier-screening program on spinal muscular atrophy births;Aharoni;Neuromuscul Disord,2020

3. Prevalence, incidence and carrier frequency of 5q–linked spinal muscular atrophy – a literature review;Verhaart;Orphanet J Rare Dis,2017

4. 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

5. Population-based analysis of survival in spinal muscular atrophy;Wijngaarde;Neurology,2020

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1. Clinical perspectives: Treating spinal muscular atrophy;Molecular Therapy;2024-08

2. A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders;Molecular Therapy;2024-07

3. Motor and neurocognitive profiles of children with symptomatic spinal muscular atrophy type 1 with two copies of SMN2 before and after treatment: a longitudinal observational study;Frontiers in Neurology;2024-02-21

4. Getting Wheels: development and evaluation of a psychoeducational resource for parents of children with a neuromuscular condition following recommendation of wheelchair equipment;Disability and Rehabilitation: Assistive Technology;2024-02-03

5. Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom;The Lancet Regional Health - Europe;2024-02