Therapeutic strategies for the treatment of spinal muscular atrophy. (Q34296140)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

title

Therapeutic strategies for the treatment of spinal muscular atrophy (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

main subject

spinal muscular atrophy

1 reference

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

author name string

Jonathan J Cherry

series ordinal

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

publication date

1 September 2012

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

Future Medicinal Chemistry

12 February 2020

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

volume

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

issue

13

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

page(s)

1733-1750

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

Identification of proximal spinal muscular atrophy carriers and patients by analysis of SMNT and SMNC gene copy number

12 February 2020

cites work

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The neurobiology of childhood spinal muscular atrophy.

21 January 2018

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Identification and characterization of a spinal muscular atrophy-determining gene

21 January 2018

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De novo rearrangements found in 2% of index patients with spinal muscular atrophy: mutational mechanisms, parental origin, mutation rate, and implications for genetic counseling

21 January 2018

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Spinal muscular atrophy: clinical classification and disease heterogeneity

21 January 2018

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Genetic mapping of chronic childhood-onset spinal muscular atrophy to chromosome 5q11.2-13.3

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The promoters of the survival motor neuron gene (SMN) and its copy (SMNc) share common regulatory elements

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Survival motor neuron SMN1 and SMN2 gene promoters: identical sequences and differential expression in neurons and non-neuronal cells

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The role of SMN in spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Survival motor neuron gene transcript analysis in muscles from spinal muscular atrophy patients.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Regulation of SMN protein stability

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Spinal muscular atrophy: from gene to therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

SMN in spinal muscular atrophy and snRNP biogenesis

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The survival motor neuron protein in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The activity of the spinal muscular atrophy protein is regulated during development and cellular differentiation.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

SMN mutants of spinal muscular atrophy patients are defective in binding to snRNP proteins.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Essential role for the SMN complex in the specificity of snRNP assembly

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Characterization of a nuclear 20S complex containing the survival of motor neurons (SMN) protein and a specific subset of spliceosomal Sm proteins

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Co-regulation of survival of motor neuron (SMN) protein and its interactor SIP1 during development and in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Survival motor neuron (SMN) protein: role in neurite outgrowth and neuromuscular maturation during neuronal differentiation and development.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The COPI vesicle complex binds and moves with survival motor neuron within axons

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Smn, the spinal muscular atrophy-determining gene product, modulates axon growth and localization of beta-actin mRNA in growth cones of motoneurons

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Knockdown of the survival motor neuron (Smn) protein in zebrafish causes defects in motor axon outgrowth and pathfinding.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Correlation between severity and SMN protein level in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Expressed cadherin pseudogenes are localized to the critical region of the spinal muscular atrophy gene

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Mapping of retrotransposon sequences in the unstable region surrounding the spinal muscular atrophy locus in 5q13.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Homozygous SMN1 deletions in unaffected family members and modification of the phenotype by SMN2.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Molecular basis of phenotypic heterogeneity in siblings with spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

A mouse model for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Neuromuscular defects and breathing disorders in a new mouse model of spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Human neural stem cells differentiate and promote locomotor recovery in spinal cord-injured mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Human embryonic stem cells differentiate into oligodendrocytes in high purity and myelinate after spinal cord transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Human embryonic stem cell-derived oligodendrocyte progenitor cell transplants remyelinate and restore locomotion after spinal cord injury

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Extensive neuronal differentiation of human neural stem cell grafts in adult rat spinal cord

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Histological and functional benefit following transplantation of motor neuron progenitors to the injured rat spinal cord

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Embryonic stem cell-derived neural stem cells improve spinal muscular atrophy phenotype in mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Donor-derived brain tumor following neural stem cell transplantation in an ataxia telangiectasia patient

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Modulating role of RNA structure in alternative splicing of a critical exon in the spinal muscular atrophy genes.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The regulation and regulatory activities of alternative splicing of the SMN gene

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

In vivo selection reveals combinatorial controls that define a critical exon in the spinal muscular atrophy genes

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Determinants of exon 7 splicing in the spinal muscular atrophy genes, SMN1 and SMN2.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Molecular and phenotypic reassessment of an infrequently used mouse model for spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Riluzole in psychiatry: a systematic review of the literature

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

A Phase 1 Trial of Riluzole in Spinal Muscular Atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Identification and characterization of cholest-4-en-3-one, oxime (TRO19622), a novel drug candidate for amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Specific antinociceptive activity of cholest-4-en-3-one, oxime (TRO19622) in experimental models of painful diabetic and chemotherapy-induced neuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Beta-lactam antibiotics offer neuroprotection by increasing glutamate transporter expression.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Beta-lactam antibiotic offers neuroprotection in a spinal muscular atrophy model by multiple mechanisms

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Induction of Nrf2 and xCT are involved in the action of the neuroprotective antibiotic ceftriaxone in vitro.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Identification of novel compounds that increase SMN protein levels using an improved SMN2 reporter cell assay.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Discovery, synthesis, and biological evaluation of novel SMN protein modulators

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Utility of survival motor neuron ELISA for spinal muscular atrophy clinical and preclinical analyses

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

A screen for regulators of survival of motor neuron protein levels

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Differential regulation of the SMN2 gene by individual HDAC proteins

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Treatment of spinal muscular atrophy by sodium butyrate

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Pilot trial of phenylbutyrate in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Valproic acid increases SMN2 expression and modulates SF2/ASF and hnRNPA1 expression in SMA fibroblast cell lines

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Valproic acid blocks excitability in SMA type I mouse motor neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Phase II open label study of valproic acid in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acid

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

In vitro and ex vivo evaluation of second-generation histone deacetylase inhibitors for the treatment of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Trichostatin A causes p53 to switch oxidative-damaged colorectal cancer cells from cell cycle arrest into apoptosis

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Deacetylase activity is required for STAT5-dependent GM-CSF functional activity in macrophages and differentiation to dendritic cells

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Stat5 constitutive activation rescues defects in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Deacetylase activity is required for recruitment of the basal transcription machinery and transactivation by STAT5.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Functional interaction of STAT5 and nuclear receptor co-repressor SMRT: implications in negative regulation of STAT5-dependent transcription

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The benzamide M344, a novel histone deacetylase inhibitor, significantly increases SMN2 RNA/protein levels in spinal muscular atrophy cells

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Mapping of DNA topoisomerase II poisons (etoposide, clerocidin) and catalytic inhibitors (aclarubicin, ICRF-187) to four distinct steps in the topoisomerase II catalytic cycle

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Inhibition of different steps of the ubiquitin system by cisplatin and aclarubicin

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Prolactin increases SMN expression and survival in a mouse model of severe spinal muscular atrophy via the STAT5 pathway.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

p38 Mitogen-activated protein kinase stabilizes SMN mRNA through RNA binding protein HuR.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Regulation of protein stability by GSK3 mediated phosphorylation

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Identification of a Maleimide-Based Glycogen Synthase Kinase-3 (GSK-3) Inhibitor, BIP-135, that Prolongs the Median Survival Time of Δ7 SMA KO Mouse Model of Spinal Muscular Atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Axon retraction and degeneration in development and disease

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The role of the Rho GTPases in neuronal development

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Fasudil improves survival and promotes skeletal muscle development in a mouse model of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Rho-kinase inactivation prolongs survival of an intermediate SMA mouse model.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Synthesis and characterization of pseudocantharidins, novel phosphatase modulators that promote the inclusion of exon 7 into the SMN (survival of motoneuron) pre-mRNA

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Modulation of SMN nuclear foci and cytoplasmic localization by its C-terminus

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Gentamicin-Induced Correction of CFTR Function in Patients with Cystic Fibrosis andCFTRStop Mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Premature stop codons involved in muscular dystrophies show a broad spectrum of readthrough efficiencies in response to gentamicin treatment.

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Translational readthrough by the aminoglycoside geneticin (G418) modulates SMN stability in vitro and improves motor function in SMA mice in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Increasing expression and decreasing degradation of SMN ameliorate the spinal muscular atrophy phenotype in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Hydroxyurea enhances SMN2 gene expression through nitric oxide release

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

The effect of hydroxyurea in spinal muscular atrophy cells and patients

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

DcpS as a therapeutic target for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Tetracyclines that promote SMN2 exon 7 splicing as therapeutics for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Clenbuterol, a beta-adrenoceptor agonist, increases relative muscle strength in orthopaedic patients

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Randomized, double-blind, placebo-controlled trial of albuterol in facioscapulohumeral dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Pilot trial of albuterol in spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

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

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Daily salbutamol in young patients with SMA type II

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Salbutamol increases survival motor neuron (SMN) transcript levels in leucocytes of spinal muscular atrophy (SMA) patients: relevance for clinical trial design

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Quantitative high-throughput screening: a titration-based approach that efficiently identifies biological activities in large chemical libraries

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

Genetic homogeneity between acute and chronic forms of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.4155%2FFMC.12.107

21 January 2018

Identifiers

DOI

10.4155/FMC.12.107

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22924510%20AND%20SRC:MED&resulttype=core&format=json

12 February 2020

1 reference