Results 231 to 240 of about 7,543 (282)
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Laboratory monitoring of nusinersen safety
Muscle & Nerve, 2021AbstractIntroductionThis retrospective study reports our tertiary care center's experience with intrathecal nusinersen administration in children and adults with spinal muscular atrophy (SMA).MethodsWe reviewed safety monitoring laboratory results and need for procedural sedation and fluoroscopy‐guidance in all SMA patients receiving nusinersen between
Natalie L. Goedeker +4 more
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Nusinersen for adults with spinal muscular atrophy
Neurological Sciences, 2023Nusinersen was effective in improving motor function and survival in infantile and childhood-onset spinal muscular atrophy (SMA), and the value of real-world experiences in adult SMA patients increase gradually. Here, we present our clinical experience in adult SMA patients treated with nusinersen according to CHERISH study.Thirty-two SMA patients ...
Doruk Arslan +5 more
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Canadian Journal of Health Technologies, 2022
CADTH recommends that Spinraza should not be reimbursed by public drug plans for the treatment of patients with type II and type III 5q spinal muscular atrophy (SMA) regardless of ambulatory status if initiated in patients older than 18 years of age. No randomized clinical trials evaluating the efficacy or safety of Spinraza in treatment ...
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CADTH recommends that Spinraza should not be reimbursed by public drug plans for the treatment of patients with type II and type III 5q spinal muscular atrophy (SMA) regardless of ambulatory status if initiated in patients older than 18 years of age. No randomized clinical trials evaluating the efficacy or safety of Spinraza in treatment ...
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Nusinersen a spinalis izomatrophia kezelésében
Ideggyógyászati szemle, 2018Until recently, the diagnosis of spinal muscular atrophy (SMA) has been associated with severe life-long motor disability in adults and with early death in infants. The new experimental therapeutic approaches of the last few years have become more and more promising, while nusinersen was approved for the treatment of SMA in December 2016 in the USA ...
Zsuzsanna Kiss +2 more
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Nusinersen: A Treatment for Spinal Muscular Atrophy
Annals of Pharmacotherapy, 2018Objective: To review the efficacy and safety of nusinersen (Spinraza) in the treatment of spinal muscular atrophy (SMA). Data Sources: An English-language literature search of PubMed and MEDLINE (1946 to June 2018) was performed using the terms nusinersen, ISIS-SMN (Rx), and spinal muscular atrophy.
Debra L Stevens +3 more
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Nusinersen for spinal muscular atrophy
Neurology, 2019Spinal muscular atrophy (SMA) is a monogenic anterior horn disease caused by mutations in the SMN1 gene leading to lack of SMN protein necessary for motor neuron functioning and survival. Phenotypic severity is modulated primarily by the amount of SMN protein produced by the paralogue SMN2 gene that differs from SMN1 by a critical single-nucleotide ...
Barry S. Russman, Emma Ciafaloni
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Nusinersen for the treatment of spinal muscular atrophy
Expert Review of Neurotherapeutics, 2017Spinal muscular atrophy (SMA) is an autosomal recessive degenerative neuromuscular disorder characterized by loss of spinal motor neurons leading to muscle weakness. This review article focuses on a novel antisense oligonucleotide treatment, first ever approved for SMA (nusinersen, SpinrazaTM) and describes the exciting journey from early ASO clinical ...
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Nusinersen: First Global Approval
Drugs, 2017Spinal muscular atrophy (SMA) is a rare autosomal recessive disorder characterized by muscle atrophy and weakness resulting from motor neuron degeneration in the spinal cord and brainstem. It is most commonly caused by insufficient levels of survival motor neuron (SMN) protein (which is critical for motor neuron maintenance) secondary to deletions or ...
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Nusinersen in the Treatment of Spinal Muscular Atrophy
2018Spinal muscular atrophy (SMA) is one of the most common genetic causes of infantile death arising due to mutations in the SMN1 gene and the subsequent loss of motor neurons. With the discovery of the intronic splicing silencer N1 (ISS-N1) as a potential target for antisense therapy, several antisense oligonucleotides (ASOs) are being developed to ...
Kara Goodkey +4 more
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Intrathecal delivery of nusinersen in individuals with complicated spines
Muscle & Nerve, 2020AbstractBackgroundThe treatment of spinal muscular atrophy (SMA) with nusinersen requires intrathecal medication administration, which can be challenging in individuals with complicated spines. This retrospective case series reviews the nusinersen treatment experience at one academic medical center with children and adults with SMA and complicated ...
Thomas G. West +3 more
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