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Effectiveness of Nusinersen in Type 1, 2 and 3 Spinal Muscular Atrophy: Croatian Real-World Data
Journal of Clinical Medicine, 2023 (1) Background: To investigate the real-world effectiveness and safety profile of nusinersen in Croatian paediatric and adult spinal muscular atrophy (SMA) patients.
Andrej Belančić +2 more
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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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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-naïve adult
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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-naïve adult
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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 ...
Emma, Ciafaloni, Barry S, Russman
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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.
Melanie K, Claborn +3 more
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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 +3 more
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