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Nusinersen

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https://www.readbyqxmd.com/read/28323809/nusinersen-spinraza-for-spinal-muscular-atrophy
#1
(no author information available yet)
No abstract text is available yet for this article.
March 27, 2017: Medical Letter on Drugs and Therapeutics
https://www.readbyqxmd.com/read/28250698/pharmaceutical-approval-update
#2
Mary Choy
Insulin degludec/liraglutide (Xultophy 100/3.6) for type-2 diabetes; rucaparib (Rubraca) for the treatment of deleterious BRCA mutation-associated ovarian cancer; and nusinersen (Spinraza) for the treatment of spinal muscular atrophy.
March 2017: P & T: a Peer-reviewed Journal for Formulary Management
https://www.readbyqxmd.com/read/28244991/oligonucleotide-therapies-for-disorders-of-the-nervous-system
#3
Olga Khorkova, Claes Wahlestedt
Oligonucleotide therapies are currently experiencing a resurgence driven by advances in backbone chemistry and discoveries of novel therapeutic pathways that can be uniquely and efficiently modulated by the oligonucleotide drugs. A quarter of a century has passed since oligonucleotides were first applied in living mammalian brain to modulate gene expression. Despite challenges in delivery to the brain, multiple oligonucleotide-based compounds are now being developed for treatment of human brain disorders by direct delivery inside the blood brain barrier (BBB)...
March 2017: Nature Biotechnology
https://www.readbyqxmd.com/read/28229309/nusinersen-first-global-approval
#4
Sheridan M Hoy
Spinal 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 mutations in the SMN1 gene. Nusinersen (SPINRAZA™) is a modified antisense oligonucleotide that binds to a specific sequence in the intron, downstream of exon 7 on the pre-messenger ribonucleic acid (pre-mRNA) of the SMN2 gene...
March 2017: Drugs
https://www.readbyqxmd.com/read/28192393/nusinersen-an-antisense-oligonucleotide-drug-for-spinal-muscular-atrophy
#5
David R Corey
No abstract text is available yet for this article.
February 13, 2017: Nature Neuroscience
https://www.readbyqxmd.com/read/28009016/motor-neuron-disease-nusinersen-potentially-effective-in-sma
#6
Louise Adams
No abstract text is available yet for this article.
February 2017: 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
#7
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, 2016: Lancet
https://www.readbyqxmd.com/read/26865511/results-from-a-phase-1-study-of-nusinersen-isis-smn-rx-in-children-with-spinal-muscular-atrophy
#8
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
#9
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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