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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…
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Keywords:
spinal muscular;
nusinersen treatment;
ethical challenges;
muscular atrophy ... See more keywords
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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…
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Keywords:
spinal muscular;
treatment;
sma;
muscular atrophy ... See more keywords
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Published in 2022 at "Medicina"
DOI: 10.3390/medicina58060766
Abstract: We read with interest the article by Abbas et al. [...].
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Keywords:
treatment spinal;
comment abbas;
nusinersen treatment;
safety efficacy ... See more keywords
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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…
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Keywords:
atrophy;
treatment;
sma type;
nusinersen treatment ... See more keywords