Efficacy and safety of nusinersen in infants with spinal muscular atrophy (SMA): Final results from the phase 3 ENDEAR study-Reference-Cited by-同舟云学术

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Efficacy and safety of nusinersen in infants with spinal muscular atrophy (SMA): Final results from the phase 3 ENDEAR study

Published:2017-06 Issue: Volume:21 Page:e14-e15
ISSN:1090-3798
Container-title:European Journal of Paediatric Neurology
language:en
Short-container-title:European Journal of Paediatric Neurology

Author:

Finkel R.,Kuntz N.,Mercuri E.,Chiriboga C.A.,Darras B.,Topaloglu H.,Montes J.,Su J.,Zhong Z.J.,Gheuens S.,Bennett C.F.,Schneider E.,Farwell W.

Publisher

Elsevier BV

Subject

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

Cited by 10 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. Down syndrome and DYRK1A overexpression: relationships and future therapeutic directions;Frontiers in Molecular Neuroscience;2024-07-24

2. Spinal muscular atrophy – the effectiveness of treatment and new therapeutic possibilities for selected groups of patients in Poland;Prospects in Pharmaceutical Sciences;2023-06-14

3. A pediatric quantitative systems pharmacology model of neurofilament trafficking in spinal muscular atrophy treated with the antisense oligonucleotide nusinersen;CPT: Pharmacometrics & Systems Pharmacology;2022-12-05

4. Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy;Advances in Therapy;2022-03-20

5. Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders;Frontiers in Genetics;2019-09-25

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