Articles with "nusinersen treatment" as a keyword



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Ethical Challenges Confronted When Providing Nusinersen Treatment for Spinal Muscular Atrophy

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Published in 2018 at "JAMA Pediatrics"

DOI: 10.1001/jamapediatrics.2017.4409

Abstract: The US Food and Drug Administration’s December 2016 approval of nusinersen for the treatment of patients with all subtypes of spinal muscular atrophy ushered in a new era for patients with spinal muscular atrophy, their… read more here.

Keywords: spinal muscular; nusinersen treatment; ethical challenges; muscular atrophy ... See more keywords
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Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy

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Published in 2021 at "Orphanet Journal of Rare Diseases"

DOI: 10.1186/s13023-021-01961-8

Abstract: Background Studies regarding the impact of (neuro)inflammation and inflammatory response following repetitive, intrathecally administered antisense oligonucleotides (ASO) in 5q-associated spinal muscular atrophy (SMA) are sparse. Increased risk of hydrocephalus in untreated SMA patients and a… read more here.

Keywords: spinal muscular; treatment; sma; muscular atrophy ... See more keywords

Comment on Abbas et al. The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. Medicina 2022, 58, 213

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Published in 2022 at "Medicina"

DOI: 10.3390/medicina58060766

Abstract: We read with interest the article by Abbas et al. [...]. read more here.

Keywords: treatment spinal; comment abbas; nusinersen treatment; safety efficacy ... See more keywords
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Nusinersen treatment of Spinal Muscular Atrophy Type 1 - results of expanded access programme in Poland.

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Published in 2021 at "Neurologia i neurochirurgia polska"

DOI: 10.5603/pjnns.a2021.0020

Abstract: AIM OF THE STUDY This study aimed to evaluate the effects of nusinersen therapy in Polish children with SMA type 1. Clinical rationale of study. Spinal muscular atrophy (SMA) is a neuromuscular disorder that is… read more here.

Keywords: atrophy; treatment; sma type; nusinersen treatment ... See more keywords