Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data-Reference-Cited by-同舟云学术

Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data

Published:2022-07 Issue: Volume:39 Page:1-10
ISSN:1090-3798
Container-title:European Journal of Paediatric Neurology
language:en
Short-container-title:European Journal of Paediatric Neurology

Author:

Erdos Judit^ORCID,Wild Claudia

Publisher

Elsevier BV

Subject

Neurology (clinical),General Medicine,Pediatrics, Perinatology and Child Health

Reference55 articles.

1. 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

2. Nusinersen (Spinraza®) modul 3 A 5q-assoziierte spinale muskelatrophie (SMA);Gemeinsamer Bundesausschuss (G-BA),2020

3. Spinraza (Nusinersen),2017

4. Zolgensma (onasemnogene abeparvovec);European Medicines Agency (EMA),2020

5. Zolgensma;Food and Drug Administration (FDA),2019

Cited by 53 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Intrathecal nusinersen treatment in a Chinese patient with spinal muscular atrophy type 3 after ventriculo-peritoneal shunt placement: A case report and review of literature;Brain and Development Case Reports;2024-12

2. Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment;Orphanet Journal of Rare Diseases;2024-09-13

3. Real-World Data in Children with Spinal Muscular Atrophy Type 1 on Long-Term Ventilation Receiving Gene Therapy: A Prospective Cohort Study;Advances in Respiratory Medicine;2024-08-28

4. Newborn Screening for Spinal Muscular Atrophy: Variations in Practice and Early Management of Infants with Spinal Muscular Atrophy in the United States;International Journal of Neonatal Screening;2024-08-16

5. Nutrition outcomes of disease modifying therapies in spinal muscular atrophy: A systematic review;Muscle & Nerve;2024-08-11