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Discovery of Risdiplam, a Selective Survival of Motor Neuron‑2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

Journal of medicinal chemistry, 2018-08, Vol.61 (15), p.6501-6517

2018

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Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study

PloS one, 2012-04, Vol.7 (4), p.e33572-e33572

2012

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SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Science (American Association for the Advancement of Science), 2014-08, Vol.345 (6197), p.688-693

2014

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Specific inhibition of myostatin activation is beneficial in mouse models of SMA therapy

Human molecular genetics, 2019-04, Vol.28 (7), p.1076-1089

2019

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Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy

Journal of medicinal chemistry, 2016-07, Vol.59 (13), p.6086-6100

2016

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Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy

Journal of medicinal chemistry, 2017-05, Vol.60 (10), p.4444-4457

2017

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Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration

PloS one, 2016-12, Vol.11 (12), p.e0167077-e0167077

2016

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miRNPs: a novel class of ribonucleoproteins containing numerous microRNAs

Genes & development, 2002-03, Vol.16 (6), p.720-728

2002

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Identification of a Human Endonuclease Complex Reveals a Link between tRNA Splicing and Pre-mRNA 3′ End Formation

Cell, 2004-04, Vol.117 (3), p.311-321

2004

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Activation of Muscle-Specific Kinase (MuSK) Reduces Neuromuscular Defects in the Delta7 Mouse Model of Spinal Muscular Atrophy (SMA)

International journal of molecular sciences, 2021-08, Vol.22 (15), p.8015

2021

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Biomarker for Spinal Muscular Atrophy: Expression of SMN in Peripheral Blood of SMA Patients and Healthy Controls

PloS one, 2015-10, Vol.10 (10), p.e0139950-e0139950

2015

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Safety, Tolerability, and Pharmacokinetics of PTC124, a Nonaminoglycoside Nonsense Mutation Suppressor, Following Single- and Multiple-Dose Administration to Healthy Male and Female Adult Volunteers

Journal of clinical pharmacology, 2007-04, Vol.47 (4), p.430-444

2007

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SMN Protein Can Be Reliably Measured in Whole Blood with an Electrochemiluminescence (ECL) Immunoassay: Implications for Clinical Trials

PloS one, 2016-03, Vol.11 (3), p.e0150640-e0150640

2016

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Nerve sprouting capacity in a pharmacologically induced mouse model of spinal muscular atrophy

Scientific reports, 2019-05, Vol.9 (1), p.7799, Article 7799

2019

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The Methylosome, a 20S Complex Containing JBP1 and pICln, Produces Dimethylarginine-Modified Sm Proteins

Molecular and Cellular Biology, 2001-12, Vol.21 (24), p.8289-8300

2001

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SMN, the Product of the Spinal Muscular Atrophy Gene, Binds Preferentially to Dimethylarginine-Containing Protein Targets

Molecular cell, 2001-05, Vol.7 (5), p.1111-1117

2001

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Detection of Arginine Dimethylated Peptides by Parallel Precursor Ion Scanning Mass Spectrometry in Positive Ion Mode

Analytical chemistry (Washington), 2003-07, Vol.75 (13), p.3107-3114

2003

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Spinal Muscular Atrophy: Disease Mechanisms and Therapy

1, 2016

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Cleavage of pre-tRNAs by the splicing endonuclease requires a composite active site

Nature, 2006-05, Vol.441 (7091), p.375-377

2006

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A Novel WD Repeat Protein Component of the Methylosome Binds Sm Proteins

The Journal of biological chemistry, 2002-03, Vol.277 (10), p.8243-8247

2002

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In vitro propagation of the prion-like state of yeast Sup35 protein

Science (American Association for the Advancement of Science), 1997-07, Vol.277 (5324), p.381-383

1997

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The SMN Complex Is Associated with snRNPs throughout Their Cytoplasmic Assembly Pathway

Molecular and Cellular Biology, 2002-09, Vol.22 (18), p.6533-6541

2002

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Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment

The Journal of clinical investigation, 2019-11, Vol.129 (11), p.4817-4831

2019

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Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers

Nature communications, 2017-11, Vol.8 (1), p.1476-13, Article 1476

2017

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Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs

Pharmacology research & perspectives, 2018-12, Vol.6 (6), p.e00447-n/a

2018

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Discovery of Risdiplam, a Selective Survival of Motor Neuron‑2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study

SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Specific inhibition of myostatin activation is beneficial in mouse models of SMA therapy

Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy

Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy

Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration

miRNPs: a novel class of ribonucleoproteins containing numerous microRNAs

Identification of a Human Endonuclease Complex Reveals a Link between tRNA Splicing and Pre-mRNA 3′ End Formation

Activation of Muscle-Specific Kinase (MuSK) Reduces Neuromuscular Defects in the Delta7 Mouse Model of Spinal Muscular Atrophy (SMA)

Biomarker for Spinal Muscular Atrophy: Expression of SMN in Peripheral Blood of SMA Patients and Healthy Controls

Safety, Tolerability, and Pharmacokinetics of PTC124, a Nonaminoglycoside Nonsense Mutation Suppressor, Following Single- and Multiple-Dose Administration to Healthy Male and Female Adult Volunteers

SMN Protein Can Be Reliably Measured in Whole Blood with an Electrochemiluminescence (ECL) Immunoassay: Implications for Clinical Trials

Nerve sprouting capacity in a pharmacologically induced mouse model of spinal muscular atrophy

The Methylosome, a 20S Complex Containing JBP1 and pICln, Produces Dimethylarginine-Modified Sm Proteins

SMN, the Product of the Spinal Muscular Atrophy Gene, Binds Preferentially to Dimethylarginine-Containing Protein Targets

Detection of Arginine Dimethylated Peptides by Parallel Precursor Ion Scanning Mass Spectrometry in Positive Ion Mode

Spinal Muscular Atrophy: Disease Mechanisms and Therapy

Cleavage of pre-tRNAs by the splicing endonuclease requires a composite active site

A Novel WD Repeat Protein Component of the Methylosome Binds Sm Proteins

In vitro propagation of the prion-like state of yeast Sup35 protein

The SMN Complex Is Associated with snRNPs throughout Their Cytoplasmic Assembly Pathway

Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment

Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers

Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs

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