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Nusinersen

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https://www.readbyqxmd.com/read/28009016/motor-neuron-disease-nusinersen-potentially-effective-in-sma
#1
Louise Adams
No abstract text is available yet for this article.
December 23, 2016: Nature Reviews. Neurology
https://www.readbyqxmd.com/read/27939059/treatment-of-infantile-onset-spinal-muscular-atrophy-with-nusinersen-a-phase-2-open-label-dose-escalation-study
#2
Richard S Finkel, Claudia A Chiriboga, Jiri Vajsar, John W Day, Jacqueline Montes, Darryl C De Vivo, Mason Yamashita, Frank Rigo, Gene Hung, Eugene Schneider, Daniel A Norris, Shuting Xia, C Frank Bennett, Kathie M Bishop
BACKGROUND: Nusinersen is a 2'-O-methoxyethyl phosphorothioate-modified antisense drug being developed to treat spinal muscular atrophy. Nusinersen is specifically designed to alter splicing of SMN2 pre-mRNA and thus increase the amount of functional survival motor neuron (SMN) protein that is deficient in patients with spinal muscular atrophy. METHODS: This open-label, phase 2, escalating dose clinical study assessed the safety and tolerability, pharmacokinetics, and clinical efficacy of multiple intrathecal doses of nusinersen (6 mg and 12 mg dose equivalents) in patients with infantile-onset spinal muscular atrophy...
December 17, 2017: Lancet
https://www.readbyqxmd.com/read/26865511/results-from-a-phase-1-study-of-nusinersen-isis-smn-rx-in-children-with-spinal-muscular-atrophy
#3
MULTICENTER STUDY
Claudia A Chiriboga, Kathryn J Swoboda, Basil T Darras, Susan T Iannaccone, Jacqueline Montes, Darryl C De Vivo, Daniel A Norris, C Frank Bennett, Kathie M Bishop
OBJECTIVE: To examine safety, tolerability, pharmacokinetics, and preliminary clinical efficacy of intrathecal nusinersen (previously ISIS-SMNRx), an antisense oligonucleotide designed to alter splicing of SMN2 mRNA, in patients with childhood spinal muscular atrophy (SMA). METHODS: Nusinersen was delivered by intrathecal injection to medically stable patients with type 2 and type 3 SMA aged 2-14 years in an open-label phase 1 study and its long-term extension. Four ascending single-dose levels (1, 3, 6, and 9 mg) were examined in cohorts of 6-10 participants...
March 8, 2016: Neurology
https://www.readbyqxmd.com/read/26823478/intrathecal-injections-in-children-with-spinal-muscular-atrophy-nusinersen-clinical-trial-experience
#4
Manon Haché, Kathryn J Swoboda, Navil Sethna, Alan Farrow-Gillespie, Alexander Khandji, Shuting Xia, Kathie M Bishop
Nusinersen (ISIS-SMNRx or ISIS 396443) is an antisense oligonucleotide drug administered intrathecally to treat spinal muscular atrophy. We summarize lumbar puncture experience in children with spinal muscular atrophy during a phase 1 open-label study of nusinersen and its extension. During the studies, 73 lumbar punctures were performed in 28 patients 2 to 14 years of age with type 2/3 spinal muscular atrophy. No complications occurred in 50 (68%) lumbar punctures; in 23 (32%) procedures, adverse events were attributed to lumbar puncture...
June 2016: Journal of Child Neurology
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