Results 201 to 210 of about 7,657 (243)

Aseptic meningoencephalitis during nusinersen therapy in a patient with type III spinal muscular atrophy: a case report.

Neuromuscular Disorders
Nusinersen, an intrathecally-administered antisense oligonucleotide for the treatment of spinal muscular atrophy (SMA), may rarely cause mild aseptic meningitis early in treatment. We report a severe late-onset aseptic brain stem meningoencephalitis in a
Lukasz Kolakowski, Olivia Zschau, Jérôme Bonzon, A. de Vere-Tyndall, M. Herwerth, C. Neuwirth, Michael Weller, Patrick Roth, B. Schreiner, V. Kana   +9 more
semanticscholar   +1 more source

Rehabilitation improves the effectiveness of nusinersen in children with type 2 spinal muscular atrophy: pNF-H and muscle MRI as potential biomarkers

Frontiers in Neurology
Introduction Rehabilitation therapy is an important approach for spinal muscular atrophy (SMA) management. Currently, rare articles introduce that the combination of nusinersen and rehabilitation yields better results in SMA patients compared to using ...
Yifan Sun, Wei Li, Xin Cui, Yang Li, Xiucheng Gao, Da-ling Fu, Xiao-ke Zhao, Tong Cao, Min Zhu   +8 more
semanticscholar   +1 more source

Development and Validation of an LC-MS/MS Assay for Quantitative Analysis of Nusinersen in Human CSF and Plasma.

Biomedical chromotography
Nusinersen is the first antisense oligonucleotide (ASO) drug approved for the treatment of spinal muscular atrophy (SMA) in China; however, its pharmacokinetics (PK) in Chinese SMA patients remains unknown.
Ting Xu, Wei Zuo, Zhuo Sun, Simin Zhang, Zhengqing Qiu, Bo Zhang, Yi Dai   +6 more
semanticscholar   +1 more source

Real-world evidence on nusinersen treatment of persons with SMA: a focused review

Journal of Neuromuscular Diseases
Nusinersen is a designer drug for spinal muscular atrophy (SMA) and was the first approved treatment for this once deadly disease. It is an antisense oligonucleotide that pairs with a specific locus of the Survival Motor Neuron 2 (SMN2) gene, to modify ...
S. Matesanz, Richard S. Finkel
semanticscholar   +1 more source

Biomarker Evolution in Pediatric SMA: Insights from CSF pNF-H Dynamics and SMN2 Copy Number During Nusinersen Therapy

Balneo and PRM Research Journal
Spinal muscular atrophy (SMA) is a severe neurodegenerative disorder caused by insufficient survival motor neuron (SMN) protein synthesis, leading to progressive motor neuron loss and debilitating symptoms.
Mihaela Bădina, G. Bejan, A. Mirea, Corina Sporea, M. Leanca, Georgiana Nicolae, Ioana Elena Cioca, M. Morcov, Angelo Pellegrini, Elena-Nicoleta Bordea   +9 more
semanticscholar   +1 more source

Nusinersen

Reactions Weekly, 2019
  +4 more sources

Respiratory Outcomes of Spinal Muscular Atrophy Patients Receiving Nusinersen in Hong Kong

Pediatric Respirology and Critical Care Medicine
Patients with spinal muscular atrophy (SMA) have significant respiratory morbidity, and their healthcare burden is high. Nusinersen, the first disease-modifying drug available for treating SMA, was administered to our patients in Hong Kong since ...
Jacqueline Hung, K. Siu, Shuk-Kuen Chau, So Lun Lee, S. Chan   +4 more
semanticscholar   +1 more source

Respiratory and Bulbar Support in Spinal Muscular Atrophy Type I Treated with Nusinersen

Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques
: Objective: The aim of this study is to assess the evolution of respiratory and feeding support in children with spinal muscular atrophy (SMA) type 1 after 24 months of nusinersen treatment.
Guillaume Gauvreau, Tarannum Behlim, Pamela Ng, V. Hodgkinson, Kathryn Selby, Jean K. Mah, Hanna Kolski, M. Castro-Codesal, M. Crone, Ed Leung, Kristina Joyal, Craig Campbell, L. McAdam, H. Gonorazky, Hugh J Mcmillan, Anna McCormick, Colleen O’Connell, M. Taillon, Maryam Oskoui   +18 more
semanticscholar   +1 more source

Assessment of Fine Motor Abilities Among Children with Spinal Muscular Atrophy Treated with Nusinersen Using a New Touchscreen Application: A Pilot Study

Children
Background/Objectives: Spinal Muscular Atrophy (SMA) is a genetic neurodegenerative disease characterized by severe muscle weakness and atrophy. Advances in disease-modifying therapies have dramatically changed the natural history of SMA and the outcome ...
Inbal Klemm, A. Danial-Saad, A. Karlin, Rya Nassar-Yassien, Iuliana Eshel, H. Levine, Tamar Steinberg, S. Aharoni   +7 more
semanticscholar   +1 more source

Risdiplam and nusinersen in spinal muscular atrophy: a descriptive real-world study on motor function outcomes in northwestern Iran.

Neuromuscular Disorders
Spinal muscular atrophy (SMA) types 2 and 3 are chronic neuromuscular disorders characterized by progressive motor impairment. Although disease-modifying therapies such as risdiplam and nusinersen have shown clinical efficacy, real-world data in ...
M. Barzegar, Bita Poorshiri, Khatereh Rezazadeh, V. Toopchizadeh, S. Shiva, A. Motamedi   +5 more
semanticscholar   +1 more source