Respiratory Follow-up of Patients Treated with Nusinersen
Turkish Thoracic Journal, 2019 Introduction and Aim: We aimed to evaluate respiratory support need in patients treated with Nusinersen and to report their respiratory follow-up during the treatment period. Material and Methods: The study included patients treated with 4 and more doses of Nusinersen.
Pınar Ergenekon +9 more
openalex +3 more sources
Switching from Nusinersen to Risdiplam: A Croatian Real-World Experience on Effectiveness and Safety [PDF]
Journal of Personalized Medicine(1) Background: Data on combination or sequential treatment of spinal muscular atrophy (SMA) with disease-modifying drugs (DMDs) are missing and the latter field is poorly understood.
Andrej Belančić +3 more
semanticscholar +2 more sources
The dynamic of changes of pNFH levels in the CSF compared with the motor scales’ scores during three years of nusinersen treatment in children with spinal muscular atrophy types 2 and 3 [PDF]
Balneo and PRM Research JournalNeurofilaments are crucial in neuronal cytoskeleton formation, influencing axonal growth and impulse modulation. This study focuses on understanding the dynamics of the phosphorylated neurofilament heavy subunit (pNFH) in pediatric spinal muscular ...
Mihaela Badina +4 more
doaj +2 more sources
Real-world analysis of the efficacy and safety of nusinersen in pediatric patients with spinal muscular atrophy [PDF]
Orphanet Journal of Rare DiseasesBackground Spinal muscular atrophy (SMA) is a rare neurodegenerative disease that significantly affects multiple systems in children. Nusinersen, the first approved treatment for SMA, enhances SMN protein production by targeting the RNA splicing site of ...
Wenjing Li +3 more
doaj +2 more sources
Neurodegeneration Biomarkers in Adult Spinal Muscular Atrophy (SMA) Patients Treated with Nusinersen [PDF]
International Journal of Molecular SciencesThe objective of this study is to evaluate biomarkers for neurodegenerative disorders in adult SMA patients and their potential for monitoring the response to nusinersen.
P. Andrés‐Benito +9 more
semanticscholar +2 more sources
Development and validation of an HILIC/MS/MS method for determination of nusinersen in rabbit plasma
HeliyonA hydrophilic interaction liquid chromatography tandem mass spectrometry (HILIC/MS/MS) method was developed and validated for the quantitative analysis of the fully phosphorothioate modified oligonucleotide nusinersen.
Xiao Zhang +6 more
doaj +2 more sources
Brain and BehaviorObjective This study was based on a retrospective clinical observational cohort study of a two‐center application of nusinersen in China to evaluate the clinical efficacy and adverse effects of nusinersen in the treatment of SMA (spinal muscular atrophy)
Dan Li +5 more
doaj +2 more sources
Unveiling the adverse events of Nusinersen in spinal muscular atrophy management based on FAERS database [PDF]
Scientific ReportsThis study aims to collect and analyze adverse event (AE) reports related to Nusinersen from the FAERS database. The study employed a combination of signal quantification techniques, including the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (
Ying Jiang +3 more
semanticscholar +2 more sources
Journal of NeurologyObjective To evaluate the effectiveness and safety of nusinersen for the treatment of 5q-spinal muscular atrophy (SMA) among Chinese pediatric patients.
Xiaoli Yao +20 more
semanticscholar +2 more sources
Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China [PDF]
BMC PediatricsBackground Nusinersen was the first approved disease modifying therapy (DMT) for spinal muscular atrophy (SMA). Intrathecal administration of nusinersen enables drug delivery directly to the central nervous system, where the motor neurons are located ...
Jing Peng +20 more
doaj +2 more sources