Results 251 to 260 of about 7,776 (285)
Some of the next articles are maybe not open access.
In Analysis
The study aimed to analyze nusinersen metabolites in the cerebrospinal fluid samples using ion-pair reversed-phase ultrahigh-performance liquid chromatography coupled with quadrupole time-of-flight mass spectrometry.
S. Studzińska +6 more
semanticscholar +1 more source
The study aimed to analyze nusinersen metabolites in the cerebrospinal fluid samples using ion-pair reversed-phase ultrahigh-performance liquid chromatography coupled with quadrupole time-of-flight mass spectrometry.
S. Studzińska +6 more
semanticscholar +1 more source
Bioanalysis
Background: The fully phosphorothioate-modified oligonucleotide (OGN) nusinersen has low ionization efficiency in the negative ion mode, resulting in a low mass spectrometry response.
Xiao Zhang +6 more
semanticscholar +1 more source
Background: The fully phosphorothioate-modified oligonucleotide (OGN) nusinersen has low ionization efficiency in the negative ion mode, resulting in a low mass spectrometry response.
Xiao Zhang +6 more
semanticscholar +1 more source
InFo Neurologie & Psychiatrie, 2018 Balneo and PRM Research Journal
Werdnig-Hoffmann disease, or type 1 Spinal Muscular Atrophy (SMA), is caused by insufficient SMN protein synthesis due to a genetic defect. Symptoms appear within the first 6 months of life, and without ventilatory support, life expectancy averages 2 ...
Mihaela Bădina +3 more
semanticscholar +1 more source
Werdnig-Hoffmann disease, or type 1 Spinal Muscular Atrophy (SMA), is caused by insufficient SMN protein synthesis due to a genetic defect. Symptoms appear within the first 6 months of life, and without ventilatory support, life expectancy averages 2 ...
Mihaela Bădina +3 more
semanticscholar +1 more source
Plecanatide, Nusinersen, and Obeticholic acid
Journal of the American Pharmacists Association, 2017Daniel A. Hussar, Deborah K. Douglas
openaire +3 more sources
Balneo and PRM Research Journal
Spinal Muscular Atrophy (SMA) is a rare neurodegenerative disease caused by insufficient synthesis of SMN protein, characterized by progressive muscle weakness, atrophy, and complications affecting the respiratory and digestive systems.
Mihaela Bădina +3 more
semanticscholar +1 more source
Spinal Muscular Atrophy (SMA) is a rare neurodegenerative disease caused by insufficient synthesis of SMN protein, characterized by progressive muscle weakness, atrophy, and complications affecting the respiratory and digestive systems.
Mihaela Bădina +3 more
semanticscholar +1 more source
Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
Nature Neuroscience, 2017Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases.
openaire +2 more sources
Neuropediatrics
Background Due to the limited data from clinical trials and real-world settings in the realm of nusinersen, there is a need for further evidence. This study seeks to assess the impact of nusinersen, when combined with standard care, on bulbar function ...
M. Gaboli +7 more
semanticscholar +1 more source
Background Due to the limited data from clinical trials and real-world settings in the realm of nusinersen, there is a need for further evidence. This study seeks to assess the impact of nusinersen, when combined with standard care, on bulbar function ...
M. Gaboli +7 more
semanticscholar +1 more source