Emerging treatment options for spinal muscular atrophy. (Q51826765)

13 April 2018

title

Emerging treatment options for spinal muscular atrophy. (English)

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13 April 2018

main subject

spinal muscular atrophy

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Barrington G Burnett

1

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13 April 2018

Thomas O Crawford

series ordinal

2

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13 April 2018

Charlotte J Sumner

series ordinal

3

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13 April 2018

publication date

1 March 2009

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13 April 2018

Current treatment options in neurology

published in

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13 April 2018

volume

11

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13 April 2018

issue

2

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13 April 2018

page(s)

90-101

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13 April 2018

Placebo-controlled trial of gabapentin in patients with amyotrophic lateral sclerosis. WALS Study Group. Western Amyotrophic Lateral Sclerosis Study Group

cites work

1 reference

https://api.crossref.org/works/10.1007%2FS11940-009-0012-X

A summary of mechanistic hypotheses of gabapentin pharmacology

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS11940-009-0012-X

Pilot trial of albuterol in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS11940-009-0012-X

Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS11940-009-0012-X

Riluzole attenuates spinal muscular atrophy disease progression in a mouse model.

21 January 2018

1 reference

12 December 2020

Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy

1 reference

12 December 2020

Identification of a cis-acting element for the regulation of SMN exon 7 splicing

1 reference

12 December 2020

A novel nuclear structure containing the survival of motor neurons protein

1 reference

12 December 2020

Consensus Statement for Standard of Care in Spinal Muscular Atrophy

1 reference

12 December 2020

The Caenorhabditis elegans orthologue of the human gene responsible for spinal muscular atrophy is a maternal product critical for germline maturation and embryonic viability.

1 reference

12 December 2020

International SMA consortium meeting. (26-28 June 1992, Bonn, Germany)

1 reference

12 December 2020

A placebo-controlled trial of gabapentin in spinal muscular atrophy

1 reference

12 December 2020

Histone deacetylase inhibitors and the promise of epigenetic (and more) treatments for cancer

1 reference

12 December 2020

SMN oligomerization defect correlates with spinal muscular atrophy severity

1 reference

12 December 2020

SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN

1 reference

12 December 2020

Daily salbutamol in young patients with SMA type II

1 reference

12 December 2020

Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts

1 reference

12 December 2020

Inactivation of the SMN complex by oxidative stress

1 reference

12 December 2020

Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3' splice site pairing

1 reference

12 December 2020

The changing natural history of spinal muscular atrophy type 1

1 reference

12 December 2020

A novel function for SMN, the spinal muscular atrophy disease gene product, in pre-mRNA splicing

1 reference

12 December 2020

Plastin 3 is a protective modifier of autosomal recessive spinal muscular atrophy

1 reference

12 December 2020

The effect of hydroxyurea in spinal muscular atrophy cells and patients

1 reference

12 December 2020

Role of gabapentin in spinal muscular atrophy: results of a multicenter, randomized Italian study

1 reference

12 December 2020

Adult neural stem cells: attempting to solve the identity crisis

1 reference

12 December 2020

Concerns about the design of clinical trials for spinal muscular atrophy

1 reference

12 December 2020

Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy

1 reference

12 December 2020

Human embryonic germ cell derivatives facilitate motor recovery of rats with diffuse motor neuron injury.

1 reference

12 December 2020

Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy

1 reference

12 December 2020

Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy

1 reference

12 December 2020

The role of histone acetylation in SMN gene expression.

1 reference

12 December 2020

Indoprofen upregulates the survival motor neuron protein through a cyclooxygenase-independent mechanism

1 reference

12 December 2020

In Vitro Restoration of Functional SMN Protein in Human Trophoblast Cells Affected by Spinal Muscular Atrophy by Small Fragment Homologous Replacement

1 reference

12 December 2020

Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients

1 reference

12 December 2020

Molecular and functional analysis of intragenic SMN1 mutations in patients with spinal muscular atrophy.

1 reference

12 December 2020

Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector.

1 reference

12 December 2020

Therapeutic benefits of cardiotrophin-1 gene transfer in a mouse model of spinal muscular atrophy

1 reference

12 December 2020

Synthesis and biological evaluation of novel 2,4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy.

1 reference

12 December 2020

Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing

1 reference

12 December 2020

Neuroprotective effects of riluzole in neurotrauma models: a review.

1 reference

12 December 2020

Treatment of spinal muscular atrophy by sodium butyrate

1 reference

12 December 2020

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

1 reference

12 December 2020

Spinal muscular atrophy: a deficiency in a ubiquitous protein; a motor neuron-specific disease

1 reference

12 December 2020

The SMN complex, an assemblyosome of ribonucleoproteins

1 reference

12 December 2020

A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels

1 reference

12 December 2020

Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition

1 reference

12 December 2020

SMN deficiency causes tissue-specific perturbations in the repertoire of snRNAs and widespread defects in splicing

1 reference

12 December 2020

Salbutamol increases SMN mRNA and protein levels in spinal muscular atrophy cells

1 reference

12 December 2020

Ribonucleoprotein assembly defects correlate with spinal muscular atrophy severity and preferentially affect a subset of spliceosomal snRNPs

1 reference

12 December 2020

Spinal muscular atrophy in the neonate

1 reference

12 December 2020

The survival motor neuron protein of Schizosacharomyces pombe. Conservation of survival motor neuron interaction domains in divergent organisms

1 reference

12 December 2020

Valproic acid increases SMN levels in spinal muscular atrophy patient cells.

1 reference

12 December 2020

A Phase 1 Trial of Riluzole in Spinal Muscular Atrophy

1 reference

12 December 2020

Diverse small-molecule modulators of SMN expression found by high-throughput compound screening: early leads towards a therapeutic for spinal muscular atrophy.

1 reference

12 December 2020

Identification and characterization of a spinal muscular atrophy-determining gene

1 reference

12 December 2020

Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos

1 reference

12 December 2020

Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells

1 reference

12 December 2020

Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs

1 reference

12 December 2020

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

1 reference

12 December 2020

Neural stem cell transplantation can ameliorate the phenotype of a mouse model of spinal muscular atrophy

1 reference

12 December 2020

A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2.

1 reference

12 December 2020

10.1007/S11940-009-0012-X

Identifiers

DOI

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13 April 2018

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13 April 2018