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Feasibility and safety of intrathecal treatment with nusinersen in adult patients with spinal muscular atrophy

Therapeutic Advances in Neurological Disorders, 2018
Background: Nusinersen is an intrathecally administered antisense oligonucleotide (ASO) and the first approved drug for the treatment of spinal muscular atrophy (SMA).
Benjamin Stolte, Saskia Bolz, Christoph Kleinschnitz   +2 more
exaly   +2 more sources

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Intrathecal delivery of nusinersen in individuals with complicated spines

Muscle & Nerve, 2020
AbstractBackgroundThe 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, Michael S. Cartwright, Zachary T. Ward, Eric P. White   +3 more
openaire   +2 more sources

Treating adults with spinal muscular atrophy with nusinersen

Journal of Neurology, Neurosurgery & Psychiatry, 2020
Nusinersen was the first drug approved to treat people with spinal muscular atrophy (SMA), based on clinical data obtained from the initial trials undertaken in infants and children.1 2 With limited evidence of the safety and efficacy of nusinersen across the spectrum of ages and severities in SMA, adult patients are now confronting uncertain ...
Michelle A Farrar, Matthew C Kiernan
openaire   +3 more sources

Safety of risdiplam in spinal muscular atrophy patients after short‐term treatment with nusinersen

Muscle and Nerve
Following the approval of risdiplam, there are more possibilities for disease‐modifying therapy (DMT) in children with spinal muscular atrophy (SMA). Non‐treatment‐naïve subjects with SMA involved in the JEWELFISH study, designed to evaluate the safety ...
Yue Yan, Yijie Feng, Liya Jiang, Jianing Jin, S. Mao   +4 more
semanticscholar   +1 more source

Phosphorylated neurofilament heavy chain in cerebrospinal fluid and plasma as a Nusinersen treatment response marker in childhood-onset SMA individuals from Serbia

Frontiers in Neurology
Introduction Biomarkers capable of reflecting disease onset and short- and long-term therapeutic effects in individuals with spinal muscular atrophy (SMA) are still an unmet need and phosphorylated neurofilament heavy chain (pNF-H) holds significant ...
M. Brkušanin, A. Kosać, V. Branković-Srećković, Kristina Jovanović, S. Perić, J. Karanović, Suzana Matijašević Joković, Nemanja Garai, J. Pešović, Dimitrije Nikolić, Z. Stevic, G. Brajušković, V. Milić-Rašić, D. Savić-Pavićević   +13 more
semanticscholar   +1 more source

Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3.

Neurology Clinical Practice
Background and Objectives Nusinersen has shown significant functional motor benefit in the milder types of spinal muscular atrophy (SMA). Less is known on the respiratory outcomes in patients with nusinersen-treated SMA.
Federica Trucco, D. Ridout, H. Weststrate, M. Scoto, A. Rohwer, G. Coratti, Marion Main, Anna G. Mayhew, Jacqueline Montes, R. de Sanctis, Marika Pane, M. Pera, V. Sansone, E. Albamonte, Adele D'Amico, Claudio Bruno, S. Messina, A. Childs, Tracey Willis, Min Ong, L. Servais, Anirban Majumdar, Imelda Hughes, C. Marini-Bettolo, D. Parasuraman, V. Gowda, Giovanni Baranello, E. Bertini, D. D. De Vivo, Basil T. Darras, J. Day, Oscar Mayer, Z. Zolkipli-Cunningham, Richard S. Finkel, Eugenio Mercuri, F. Muntoni   +35 more
semanticscholar   +1 more source

Effect of Discontinuation of Nusinersen Treatment in Long-Standing SMA3

Journal of Neuromuscular Diseases, 2021
Background: Spinal muscular atrophy is an autosomal recessive neuromuscular disease leading to ongoing degeneration of anterior horn cells in the spinal cord. Nusinersen is the first approved treatment for the condition, an intrathecally administered antisense oligonucleotide. It modulates pre-RNA splicing of the SMN2 gene and increases full-length SMN
Maggie C. Walter, Peter Reilich, Miriam Hiebeler, Angela Abicht   +3 more
openaire   +3 more sources

Analytical Quality by Design–Based Approach for the Assay Method Development of Nusinersen Using Liquid Chromatography–High‐Resolution Mass Spectrometry

Separation Science Plus
The quality assessment of oligonucleotide possesses challenges due to non‐distinguishable structurally similar impurities. These impurities are difficult to separate from the drug, and the assay method as per regulatory requirements must quantify only ...
Vijay S. Bhalekar, Ravi P. Shah
semanticscholar   +1 more source

Impact of Nusinersen on Neurofilament, Creatinine Levels, and Motor Function in Pediatric Spinal Muscular Atrophy Rehabilitation: A Biomarker Analysis

Balneo and PRM Research Journal
Background: Spinal amyotrophy is a rare, neurodegenerative disease, with progressive evolution, disabling until death in severe forms, but for which 3 disease-modifying drugs have recently been approved (in the last 8 years).
Mihaela Bădina, Corina Sporea, G. Bejan, A. Mirea, Daniela Adriana Ion   +4 more
semanticscholar   +1 more source

Ultrasound-guided cervical puncture for nusinersen administration in adolescents

Pediatric Radiology, 2018
Spinal muscular atrophy (SMA) is an autosomal-recessive disease affecting motor neurons and is the most common genetic cause of death in infants. Intrathecal nusinersen is the only therapy approved by the U.S. Food and Drug Administration for SMA.
Timothy Lotze, Timothy Lotze, Carlos B Ortiz, Alex Chau, Alex Chau, Kamlesh Kukreja, Kamlesh Kukreja   +6 more
openaire   +3 more sources