Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps

Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps

Spinal muscular atrophy (SMA) is a recessive disorder caused by a mutation in the survival motor neuron 1 gene (SMN1); it affects 1 in 11 000 newborn infants. The most severe and most common form, type 1 SMA, is associated with early mortality in most cases and severe disability in survivors. Nusine...

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Bibliografiske detaljer
Main Authors:	Gidaro, T, Servais, L
Format:	Journal article
Sprog:	English
Udgivet:	Wiley 2018

Lignende værker

Sitting in patients with spinal muscular atrophy type 1 treated with nusinersen
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Udgivet: (2017)

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Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy
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Udgivet: (2021-07-01)

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af: Aragon-Gawinska, K, et al.
Udgivet: (2018)

Nusinersen versus sham control in later-onset spinal muscular atrophy
af: Mercuri, E, et al.
Udgivet: (2018)

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Udgivet: (2022-06-01)

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Udgivet: (2021-08-01)

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Udgivet: (2020-01-01)

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Udgivet: (2024-07-01)

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Udgivet: (2024-08-01)

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Udgivet: (2020-01-01)

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Udgivet: (2020)

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Udgivet: (2025-01-01)

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Udgivet: (2021-03-01)

Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy
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Udgivet: (2022-10-01)

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af: Praveen Thokala, et al.
Udgivet: (2020-10-01)

Safety, Tolerability, and Effect of Nusinersen in Non-ambulatory Adults With Spinal Muscular Atrophy
af: Bakri Elsheikh, et al.
Udgivet: (2021-04-01)

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Udgivet: (2024-07-01)

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af: Asma AlTawari, et al.
Udgivet: (2024-06-01)

Clinical and Electrophysiological Changes in Pediatric Spinal Muscular Atrophy after 2 Years of Nusinersen Treatment
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Udgivet: (2022-09-01)

Intrathecal administration of nusinersen for spinal muscular atrophy: report of three cases with severe spinal deformity
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Udgivet: (2020-04-01)

Real-world analysis of the efficacy and safety of nusinersen in pediatric patients with spinal muscular atrophy
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Udgivet: (2025-02-01)

Effectiveness of Nusinersen in Adolescents and Adults with Spinal Muscular Atrophy: Systematic Review and Meta-analysis
af: Tim Hagenacker, et al.
Udgivet: (2024-09-01)

Unveiling the adverse events of Nusinersen in spinal muscular atrophy management based on FAERS database
af: Ying Jiang, et al.
Udgivet: (2024-07-01)

Safety analysis of laboratory parameters in paediatric patients with spinal muscular atrophy treated with nusinersen
af: Xiaomei Zhu, et al.
Udgivet: (2024-07-01)

Persistent neuromuscular junction transmission defects in adults with spinal muscular atrophy treated with nusinersen
af: Tristan Weaver, et al.
Udgivet: (2021-09-01)

Effects of nusinersen on motor function in children with spinal muscular atrophy: a retrospective study
af: Yuyi Chen, et al.
Udgivet: (2024-07-01)

Quality of Life Outcomes According to Differential Nusinersen Exposure in Pediatric Spinal Muscular Atrophy
af: Meaghann S. Weaver, et al.
Udgivet: (2021-07-01)

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af: Can Özütemiz, et al.
Udgivet: (2020-11-01)

Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy
af: Marika Pane, et al.
Udgivet: (2022-03-01)

Experience of Nusinersen in children with proximal spinal muscular atrophy 5q in Moscow region
af: M. V. Panteleeva
Udgivet: (2022-12-01)

Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives
af: Dangouloff T, et al.
Udgivet: (2019-10-01)

A Semi‐Mechanistic Population Pharmacokinetic Model of Nusinersen: An Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy
af: Konstantinos Biliouris, et al.
Udgivet: (2018-09-01)