(Q38115148)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23775131%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

title

Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy (English)

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3 March 2020

1

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3 March 2020

Francesca Magri

2

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3 March 2020

Irene Faravelli

5

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3 March 2020

Stefania Corti

6

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23775131%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

Nereo Bresolin

7

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3 March 2020

Giacomo P Comi

8

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23775131%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

author name string

Simona Brajkovic

3

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23775131%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

Chiara Zanetta

4

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3 March 2020

publication date

18 June 2013

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Cellular and Molecular Life Sciences

3 March 2020

published in

1 reference

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3 March 2020

volume

70

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23775131%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

issue

23

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3 March 2020

page(s)

4585-4602

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https://scigraph.springernature.com/pub.10.1007/s00018-013-1396-z

3 March 2020

exact match

0 references

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Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials

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Cardiomyopathy of Duchenne muscular dystrophy: current understanding and future directions

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Genetic predictors and remodeling of dilated cardiomyopathy in muscular dystrophy

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Pathophysiology and therapy of cardiac dysfunction in Duchenne muscular dystrophy

21 January 2018

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Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

21 January 2018

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Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care

21 January 2018

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Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy.

21 January 2018

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Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study.

21 January 2018

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The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy.

21 January 2018

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Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy

21 January 2018

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24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy

21 January 2018

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A critical review of functional assessment tools for upper limbs in Duchenne muscular dystrophy.

21 January 2018

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Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy.

21 January 2018

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The PedsQL in pediatric patients with Duchenne muscular dystrophy: feasibility, reliability, and validity of the Pediatric Quality of Life Inventory Neuromuscular Module and Generic Core Scales

21 January 2018

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Abnormal distribution of inositol 1,4,5-trisphosphate receptors in human muscle can be related to altered calcium signals and gene expression in Duchenne dystrophy-derived cells.

21 January 2018

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Applications of metabolomics and proteomics to the mdx mouse model of Duchenne muscular dystrophy: lessons from downstream of the transcriptome

21 January 2018

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Identification of disease specific pathways using in vivo SILAC proteomics in dystrophin deficient mdx mouse.

21 January 2018

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Upregulation of chemokines and their receptors in Duchenne muscular dystrophy: potential for attenuation of myofiber necrosis.

21 January 2018

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Identification of muscle-specific microRNAs in serum of muscular dystrophy animal models: promising novel blood-based markers for muscular dystrophy

21 January 2018

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Expression analysis in multiple muscle groups and serum reveals complexity in the microRNA transcriptome of the mdx mouse with implications for therapy

21 January 2018

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miRNAs as serum biomarkers for Duchenne muscular dystrophy

21 January 2018

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Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion

21 January 2018

1 reference

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iNOS ablation does not improve specific force of the extensor digitorum longus muscle in dystrophin-deficient mdx4cv mice

21 January 2018

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Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

21 January 2018

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Muscle histology vs MRI in Duchenne muscular dystrophy

21 January 2018

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Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

21 January 2018

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PTC124 targets genetic disorders caused by nonsense mutations

21 January 2018

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Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

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Genetic treatments in muscular dystrophies

21 January 2018

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In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

21 January 2018

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Impending therapies for Duchenne muscular dystrophy

21 January 2018

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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

21 January 2018

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Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense

21 January 2018

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Multiple exon skipping strategies to by-pass dystrophin mutations

21 January 2018

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Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

21 January 2018

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Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model

21 January 2018

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Systemic administration of PRO051 in Duchenne's muscular dystrophy

21 January 2018

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Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.

21 January 2018

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Proteomic profiling of antisense-induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm.

21 January 2018

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Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

21 January 2018

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In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse

21 January 2018

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Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

21 January 2018

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Exon skipping quantification by quantitative reverse-transcription polymerase chain reaction in Duchenne muscular dystrophy patients treated with the antisense oligomer eteplirsen

21 January 2018

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Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.

21 January 2018

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Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy

21 January 2018

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Chemical treatment enhances skipping of a mutated exon in the dystrophin gene

21 January 2018

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Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo

21 January 2018

1 reference

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Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy

21 January 2018

1 reference

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Comparison between gentamycin and exon skipping treatments to restore ryanodine receptor subtype 2 functions in mdx mouse duodenum myocytes.

21 January 2018

1 reference

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Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.

21 January 2018

1 reference

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Correction of the dystrophic phenotype by in vivo targeting of muscle progenitor cells

21 January 2018

1 reference

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Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice

21 January 2018

1 reference

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Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy

21 January 2018

1 reference

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A canine minidystrophin is functional and therapeutic in mdx mice

21 January 2018

1 reference

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microRNA-206 promotes skeletal muscle regeneration and delays progression of Duchenne muscular dystrophy in mice

21 January 2018

1 reference

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Loss of miR-29 in myoblasts contributes to dystrophic muscle pathogenesis

21 January 2018

1 reference

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Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade.

21 January 2018

1 reference

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Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy

21 January 2018

1 reference

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Treatment of Duchenne muscular dystrophy with ciclosporin A: a randomised, double-blind, placebo-controlled multicentre trial

21 January 2018

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Stage-specific modulation of skeletal myogenesis by inhibitors of nuclear deacetylases.

21 January 2018

1 reference

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Deacetylase inhibitors increase muscle cell size by promoting myoblast recruitment and fusion through induction of follistatin

21 January 2018

1 reference

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Isolation of nitric oxide synthetase, a calmodulin-requiring enzyme

21 January 2018

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Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy

21 January 2018

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Nitric oxide sustains long-term skeletal muscle regeneration by regulating fate of satellite cells via signaling pathways requiring Vangl2 and cyclic GMP.

21 January 2018

1 reference

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A dual acting compound releasing nitric oxide (NO) and ibuprofen, NCX 320, shows significant therapeutic effects in a mouse model of muscular dystrophy

21 January 2018

1 reference

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Daily administration of phosphodiesterase type 5 inhibitors for urological and nonurological indications

21 January 2018

1 reference

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Primary role of functional ischemia, quantitative evidence for the two-hit mechanism, and phosphodiesterase-5 inhibitor therapy in mouse muscular dystrophy

21 January 2018

1 reference

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Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy.

21 January 2018

1 reference

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Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance

21 January 2018

1 reference

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Role of IGF-I in skeletal muscle mass maintenance

21 January 2018

1 reference

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Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00018-013-1396-Z

Stem cell-mediated transfer of a human artificial chromosome ameliorates muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00018-013-1396-Z

Partial dysferlin reconstitution by adult murine mesoangioblasts is sufficient for full functional recovery in a murine model of dysferlinopathy.

21 January 2018

1 reference

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North Star Ambulatory Assessment, 6-minute walk test and timed items in ambulant boys with Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00018-013-1396-Z

Human skin-derived stem cells migrate throughout forebrain and differentiate into astrocytes after injection into adult mouse brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00018-013-1396-Z

IGF-I treatment improves the functional properties of fast- and slow-twitch skeletal muscles from dystrophic mice

21 January 2018

1 reference

12 December 2020

Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up

1 reference

12 December 2020

Nitric oxide donor and non steroidal anti inflammatory drugs as a therapy for muscular dystrophies: Evidence from a safety study with pilot efficacy measures in adult dystrophic patients

1 reference

12 December 2020

Markers of degeneration and regeneration in Duchenne muscular dystrophy

1 reference

12 December 2020

Proteomic analysis of dystrophic muscle

1 reference

12 December 2020

Treatment of dystrophinopathic cardiomyopathy: review of the literature and personal results.

1 reference

12 December 2020

Mild and severe muscular dystrophy associated with deletions in Xp21 of the human X chromosome

1 reference

12 December 2020

Exploring the molecular basis for variability among patients with Becker muscular dystrophy: dystrophin gene and protein studies

1 reference

12 December 2020

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.

1 reference

12 December 2020

Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy

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12 December 2020

The effect of 6-thioguanine on alternative splicing and antisense-mediated exon skipping treatment for duchenne muscular dystrophy

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12 December 2020

Antisense oligonucleotides: basic concepts and mechanisms

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12 December 2020

Glucocorticoid corticosteroids for Duchenne muscular dystrophy

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12 December 2020

Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial

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12 December 2020

Local dystrophin restoration with antisense oligonucleotide PRO051

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12 December 2020

Autologous Transplantation of Muscle-Derived CD133+ Stem Cells in Duchenne Muscle Patients

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12 December 2020

A motor function measure for neuromuscular diseases. Construction and validation study

1 reference

12 December 2020

Validity of the EK scale: a functional assessment of non-ambulatory individuals with Duchenne muscular dystrophy or spinal muscular atrophy

1 reference

12 December 2020

LTBP4 genotype predicts age of ambulatory loss in Duchenne muscular dystrophy

1 reference

12 December 2020

Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands

1 reference

12 December 2020

Becker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patients

1 reference

12 December 2020

Histone deacetylase inhibitors in the treatment of muscular dystrophies: epigenetic drugs for genetic diseases.

1 reference

12 December 2020

10.1007/S00018-013-1396-Z

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23775131%20AND%20SRC:MED&resulttype=core&format=json

3 March 2020

1 reference