Keyword

nusinersen

Description: Nusinersen is an antisense oligonucleotide drug used to treat spinal muscular atrophy by modifying SMN2 gene splicing.

Primary topic: Neurogenetic and Muscular Disorders Research (87%)

Wikidata ID: wiki/Q25105466

OpenAlex ID: keywords/nusinersen

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

2017 · Richard S. Finkel, Eugenio Mercuri, et al. · New England Journal of Medicine · 2,326

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Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

2018 · Eugenio Mercuri, Basil T. Darras, et al. · New England Journal of Medicine · 1,428

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Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

2016 · Richard S Finkel, Claudia A Chiriboga, et al. · The Lancet · 1,019

Antisense technology: an overview and prospectus

2021 · Stanley T. Crooke, Brenda F. Baker, et al. · Nature Reviews Drug Discovery · 764

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

2019 · Darryl C. De Vivo, Enrico Bertini, et al. · Neuromuscular Disorders · 631

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Results from a phase 1 study of nusinersen (ISIS-SMN Rx ) in children with spinal muscular atrophy

2016 · Claudia A. Chiriboga, Kathryn J. Swoboda, et al. · Neurology · 613

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Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

2019 · C. Frank Bennett, Adrian R. Krainer, et al. · Annual Review of Neuroscience · 351

Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study

2020 · Tim Hagenacker, Claudia D Wurster, et al. · The Lancet Neurology · 331

Nusinersen: First Global Approval

2017 · Sheridan M. Hoy · Drugs · 298

Nusinersen in later-onset spinal muscular atrophy

2019 · Basil T. Darras, Claudia A. Chiriboga, et al. · Neurology · 262

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Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

2017 · David R Corey · Nature Neuroscience · 240

Advances in therapy for spinal muscular atrophy: promises and challenges

2018 · Ewout J. N. Groen, Kevin Talbot, et al. · Nature Reviews Neurology · 235

Intrathecal Injections in Children With Spinal Muscular Atrophy

2016 · Manon Haché, Kathryn J. Swoboda, et al. · Journal of Child Neurology · 208

Neurofilament as a potential biomarker for spinal muscular atrophy

2019 · Basil T. Darras, Thomas O. Crawford, et al. · Annals of Clinical and Translational Neurology · 204

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Safety and Treatment Effects of Nusinersen in Longstanding Adult 5q-SMA Type 3 – A Prospective Observational Study

2019 · Maggie C. Walter, Stephan Wenninger, et al. · Journal of Neuromuscular Diseases · 202

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FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

2017 · Annemieke Aartsma-Rus · Nucleic Acid Therapeutics · 196

Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment

2019 · Daniel M. Ramos, Constantin d’Ydewalle, et al. · Journal of Clinical Investigation · 193

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One Year of Newborn Screening for SMA – Results of a German Pilot Project

2019 · Katharina Vill, Heike Kölbel, et al. · Journal of Neuromuscular Diseases · 187

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Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3

2020 · Lorenzo Maggi, Luca Bello, et al. · Journal of Neurology Neurosurgery & Psychiatry · 179

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Oligonucleotide therapies for disorders of the nervous system

2017 · Olga Khorkova, Claes Wahlestedt · Nature Biotechnology · 179

ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy

2017 · Eric W. Ottesen · Translational Neuroscience · 173

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Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps

2018 · Teresa Gidaro, Laurent Servais · Developmental Medicine & Child Neurology · 172

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Spinal Muscular Atrophy: Mutations, Testing, and Clinical Relevance

2021 · Melissa C Keinath, Devin E Prior, et al. · The Application of Clinical Genetics · 171

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How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy

2017 · N N Singh, M D Howell, et al. · Gene Therapy · 171

Nusinersen for the treatment of spinal muscular atrophy

2017 · Claudia A. Chiriboga · Expert Review of Neurotherapeutics · 164

Works count: 2,421

Citation count: 31,790