(Q34172502)

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Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development

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scholarly article

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Europe PubMed Central

PubMed publication ID

31 July 2017

Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development (English)

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

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spinal muscular atrophy

1 reference

based on heuristic

inferred from title

muscular atrophy

1 reference

based on heuristic

inferred from title

1 reference

based on heuristic

inferred from title

author name string

Rebecca M Pruss

1

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

Marc Giraudon-Paoli

2

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

Svetlana Morozova

3

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

Patrick Berna

4

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

Jean-Louis Abitbol

5

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

Thierry Bordet

6

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

publication date

1 September 2010

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

Future Medicinal Chemistry

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

2

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

9

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

1429-1440

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Challenges and opportunities in clinical trials for spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Identification and characterization of a spinal muscular atrophy-determining gene

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Spinal muscular atrophies: recent insights and impact on molecular diagnosis

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

The survival motor neuron protein in spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Correlation between severity and SMN protein level in spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

A mouse model for spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

The SMN complex, an assemblyosome of ribonucleoproteins

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Specific interaction of Smn, the spinal muscular atrophy determining gene product, with hnRNP-R and gry-rbp/hnRNP-Q: a role for Smn in RNA processing in motor axons?

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Restoring Bcl-x(L) levels benefits a mouse model of spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Survival motor neuron gene 2 silencing by DNA methylation correlates with spinal muscular atrophy disease severity and can be bypassed by histone deacetylase inhibition

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Neuromuscular defects in a Drosophila survival motor neuron gene mutant.

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

A Drosophila melanogaster model of spinal muscular atrophy reveals a function for SMN in striated muscle.

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Modeling spinal muscular atrophy in Drosophila

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Deletion of murine SMN exon 7 directed to skeletal muscle leads to severe muscular dystrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Olesoxime (cholest-4-en-3-one, oxime): analgesic and neuroprotective effects in a rat model of painful peripheral neuropathy produced by the chemotherapeutic agent, paclitaxel

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Olesoxime (TRO19622): A Novel Mitochondrial-Targeted Neuroprotective Compound

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Olesoxime prevents microtubule-targeting drug neurotoxicity: selective preservation of EB comets in differentiated neuronal cells.

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

The role of histone acetylation in SMN gene expression.

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Treatment of spinal muscular atrophy by sodium butyrate

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

DcpS as a therapeutic target for spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Pyranmycins, a novel class of aminoglycosides with improved acid stability: the SAR of D-pyranoses on ring III of pyranmycin.

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Correction of disease-associated exon skipping by synthetic exon-specific activators

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy.

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Evolving concepts on human SMN pre-mRNA splicing.

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Natural history of denervation in SMA: relation to age, SMN2 copy number, and function

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Different atrophy-hypertrophy transcription pathways in muscles affected by severe and mild spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

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

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Reliability of the Hammersmith functional motor scale for spinal muscular atrophy in a multicentric study

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Genetic homogeneity between acute and chronic forms of spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.4155%2FFMC.10.228

21 January 2018

Identifiers

10.4155/FMC.10.228

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

31 July 2017

ResearchGate publication ID

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