Development and Validation of an Ion-Pair Reverse-Phase High-Performance Liquid Chromatography-Electrospray Ionization Mass Spectrometry Method for Determination of Purity of Nusinersen for Quality Control of Drug Substance or Drug Product. [PDF]
Samoilov M, Zubareva E, Degterev M.
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Cognitive and neurodevelopmental outcomes in spinal muscular atrophy: a scoping review. [PDF]
Gnazzo M +8 more
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Unbiased bioinformatic and clinical analysis identifies glial fibrillary acidic protein as a potential biomarker in spinal muscular atrophy. [PDF]
Soares de Oliveira KL +12 more
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Delayed diagnosis and genetic testing in spinal muscular atrophy: a case series. [PDF]
Guo X +7 more
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Impact of Intermittent Positive Pressure Breathing on Thoracic Growth in Children With Type 1 Spinal Muscular Atrophy. [PDF]
Thébault C +9 more
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Serum p-tau181 and p-tau217 levels in adolescents and adults with 5q spinal muscular atrophy. [PDF]
Stapf CD +15 more
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Administration practice and adherence of nusinersen in adults with 5q-spinal muscular atrophy in China: an ambispective multicenter study. [PDF]
Yao X +15 more
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Nusinersen effectiveness and safety in children with type II/III 5q-spinal muscular atrophy: Second interim analysis from a Chinese disease registry. [PDF]
Wang X +25 more
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Longitudinal motor function and biomarker correlates in treated adult spinal muscular atrophy: a single-center cohort study. [PDF]
Yano C +15 more
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