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

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

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

http://www.wikidata.org/entity/Q36661721

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Utility of Induced Pluripotent Stem Cells for the Study and Treatment of Genetic Diseases: Focus on Childhood Neurological Disorders Integrated Safety Assessment of 2'-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers Emerging therapies and challenges in spinal muscular atrophy Pharmacology, pharmacokinetics, and metabolism of the DNA-decoy AYX1 for the prevention of acute and chronic post-surgical pain Delivery is key: lessons learnt from developing splice-switching antisense therapies Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells.Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts Big win possible for Ionis/Biogen antisense drug in muscular atrophy.ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.A Comparative Study of SMN Protein and mRNA in Blood and Fibroblasts in Patients with Spinal Muscular Atrophy and Healthy Controls Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration.AMPA GluA1-flip targeted oligonucleotide therapy reduces neonatal seizures and hyperexcitability Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool.Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy.Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience.Splice-switching antisense oligonucleotides as therapeutic drugs Altered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3.Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders.The clinical landscape for SMA in a new therapeutic era.Longitudinal characterization of biomarkers for spinal muscular atrophy.Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.Population Pharmacokinetics of Nusinersen in the Cerebral Spinal Fluid and Plasma of Pediatric Patients With Spinal Muscular Atrophy Following Intrathecal Administrations.Non-coding RNAs as drug targets.Advances in modeling and treating spinal muscular atrophy Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy.Nusinersen: First Global Approval.The chemical evolution of oligonucleotide therapies of clinical utility.New treatments for serious conditions: ethical implications.How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy Cure SMA and our patient community celebrate the first approved drug for SMA.Splice-Switching Therapy for Spinal Muscular Atrophy.Developing multidisciplinary clinics for neuromuscular care and research.Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases.Pharmacological therapeutics in Friedreich ataxia: the present state.

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

http://www.wikidata.org/entity/Q36661721

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2016 nî lūn-bûn

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Results from a phase 1 study o ...... with spinal muscular atrophy.

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Results from a phase 1 study o ...... with spinal muscular atrophy.

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Results from a phase 1 study o ...... with spinal muscular atrophy.

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C Frank Bennett

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Claudia A Chiriboga

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Daniel A Norris

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Darryl C De Vivo

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Jacqueline Montes

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Kathie M Bishop

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Susan T Iannaccone

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P2860

SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy

An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients

Validation of the Expanded Hammersmith Functional Motor Scale in spinal muscular atrophy type II and III

Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy

Identification and characterization of a spinal muscular atrophy-determining gene

Spinal muscular atrophy.

Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.

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Kathryn J Swoboda

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26865511

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spinal muscular atrophy

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