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Cognitive and Behavioral Profile of Egyptian Children with Duchenne Muscular Dystrophy.
- Published in:
- Alexandria Journal of Pediatrics, 2023, v. 36, n. 1, p. 51, doi. 10.4103/ajop.ajop_12_23
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- Publication type:
- Article
Make or break for first splice-modulating agents.
- Published in:
- Nature Reviews Drug Discovery, 2013, v. 12, n. 11, p. 813, doi. 10.1038/nrd4151
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- Publication type:
- Article
Market watch: Upcoming market catalysts in Q4 2013.
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- Nature Reviews Drug Discovery, 2013, v. 12, n. 10, p. 731, doi. 10.1038/nrd4136
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- Publication type:
- Article
RNA therapeutics: beyond RNA interference and antisense oligonucleotides.
- Published in:
- Nature Reviews Drug Discovery, 2012, v. 11, n. 2, p. 125, doi. 10.1038/nrd3625
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- Publication type:
- Article
Targeting RNA to treat neuromuscular disease.
- Published in:
- 2011
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- Publication type:
- journal article
Deal watch: Acceleron and Shire to target the activin receptor pathway for muscular disorders.
- Published in:
- 2010
- Publication type:
- journal article
Targeting RNA: an emerging hope for treating muscular dystrophy.
- Published in:
- Nature Reviews Drug Discovery, 2009, v. 8, n. 12, p. 917, doi. 10.1038/nrd3069
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- Publication type:
- Article
Fixing the leak.
- Published in:
- 2009
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- Publication type:
- journal article
Genetic neuromuscular disorders: living the era of a therapeutic revolution. Part 2: diseases of motor neuron and skeletal muscle.
- Published in:
- 2019
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- Publication type:
- journal article
Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges.
- Published in:
- Journal of Personalized Medicine, 2018, v. 8, n. 4, p. 41, doi. 10.3390/jpm8040041
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- Publication type:
- Article
Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy.
- Published in:
- Journal of Personalized Medicine, 2018, v. 8, n. 4, p. 38, doi. 10.3390/jpm8040038
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- Publication type:
- Article
Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects.
- Published in:
- Clinical Pharmacology in Drug Development, 2019, v. 8, n. 2, p. 172, doi. 10.1002/cpdd.645
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- Publication type:
- Article
Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy.
- Published in:
- Degenerative Neurological & Neuromuscular Disease, 2016, v. 6, p. 37, doi. 10.2147/DNND.S71808
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- Publication type:
- Article
X-Linked Dilated Cardiomyopathy: A Cardiospecific Phenotype of Dystrophinopathy.
- Published in:
- Pharmaceuticals (14248247), 2015, v. 8, n. 2, p. 303, doi. 10.3390/ph8020303
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- Publication type:
- Article
Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.
- Published in:
- Pharmaceuticals (14248247), 2013, v. 6, n. 7, p. 813, doi. 10.3390/ph6070813
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- Publication type:
- Article
Using CRISPR/Cas9 System to Knock out Exon 48 in DMD Gene.
- Published in:
- Avicenna Journal of Medical Biotechnology, 2021, v. 13, n. 2, p. 54, doi. 10.18502/ajmb.v13i2.5517
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- Publication type:
- Article
Repairing the genomic defect in muscular dystrophy.
- Published in:
- Clinical Genetics, 2000, v. 58, n. 4, p. 265, doi. 10.1034/j.1399-0004.2000.580403.1.x
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- Publication type:
- Article
Fracture in Duchenne Muscular Dystrophy.
- Published in:
- Journal of Child Neurology, 2016, v. 31, n. 9, p. 1181, doi. 10.1177/0883073816650034
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- Publication type:
- Article
DYSTROFIA MIĘŚNIOWA TYPU DUCHENNE’A ZE SZCZEGÓLNYM UWZGLĘDNIENIEM ZMIAN W UKŁADZIE SERCOWO-NACZYNIOWYM ORAZ ASPEKTÓW GENETYCZNYCH.
- Published in:
- Przeglad Pediatryczny, 2012, v. 42, n. 4, p. 200
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- Publication type:
- Article
Naproxcinod shows significant advantages over naproxen in the mdx model of Duchenne Muscular Dystrophy.
- Published in:
- Orphanet Journal of Rare Diseases, 2015, v. 10, n. 1, p. 1, doi. 10.1186/s13023-015-0311-0
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- Publication type:
- Article
A comprehensive database of Duchenne and Becker muscular dystrophy patients (0-18 years old) in East China.
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- Orphanet Journal of Rare Diseases, 2015, v. 10, n. 1, p. 1, doi. 10.1186/s13023-014-0220-7
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- Publication type:
- Article
Interplay of IKK/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy.
- Published in:
- 2007
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- Publication type:
- journal article
Non-ambulatory Duchenne muscular dystrophy: observations, interventions, and outcomes of a single case.
- Published in:
- Paediatrica Indonesiana, 2022, v. 62, n. 3, p. 208, doi. 10.14238/pi62.3.2022.208-16
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- Publication type:
- Article
Therapeutic drug monitoring of vancomycin in a patient with Duchenne muscular dystrophy (DMD): A case report.
- Published in:
- 2016
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- Publication type:
- Case Study
New variants, challenges and pitfalls in DMD genotyping: implications in diagnosis, prognosis and therapy.
- Published in:
- Journal of Human Genetics, 2014, v. 59, n. 8, p. 454, doi. 10.1038/jhg.2014.54
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- Publication type:
- Article
The extraocular muscle stem cell niche is resistant to ageing and disease.
- Published in:
- Frontiers in Aging Neuroscience, 2014, v. 6, p. 1, doi. 10.3389/fnagi.2014.00328
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- Publication type:
- Article
Epigenetic Reprogramming of Muscle Progenitors: Inspiration for Clinical Therapies.
- Published in:
- Thrombosis, 2015, p. 1, doi. 10.1155/2016/6093601
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- Publication type:
- Article
Nanopatterned Human iPSC-Based Model of a Dystrophin-Null Cardiomyopathic Phenotype.
- Published in:
- Cellular & Molecular Bioengineering, 2015, v. 8, n. 3, p. 320, doi. 10.1007/s12195-015-0413-8
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- Publication type:
- Article
Duchenne muscular dystrophy: genome editing gives new hope for treatment.
- Published in:
- 2018
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- Publication type:
- journal article
EMA reconsiders 'read-through' drug against Duchenne muscular dystrophy following appeal.
- Published in:
- Nature Biotechnology, 2014, v. 32, n. 8, p. 706, doi. 10.1038/nbt0814-706
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- Publication type:
- Article
Anti-stress therapy for muscular dystrophy.
- Published in:
- Nature Biotechnology, 2012, v. 30, n. 6, p. 508, doi. 10.1038/nbt.2275
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- Publication type:
- Article
Effect of yoga as an add-on therapy in the modulation of heart rate variability in children with duchenne muscular dystrophy.
- Published in:
- International Journal of Yoga, 2019, v. 12, n. 1, p. 55, doi. 10.4103/ijoy.IJOY_12_18
- Publication type:
- Article
'It is easier to not allow them to see your disability straight away, to see you as a person': An Interpretative Phenomenological Analysis of video gaming from the perspectives of men with Duchenne Muscular Dystrophy.
- Published in:
- Palliative Medicine, 2023, v. 37, n. 7, p. 1016, doi. 10.1177/02692163231172246
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- Publication type:
- Article
Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.
- Published in:
- Nature Medicine, 2015, v. 21, n. 3, p. 270, doi. 10.1038/nm.3765
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- Publication type:
- Article
Exon-skipping drug pulls ahead in muscular dystrophy field.
- Published in:
- Nature Medicine, 2012, v. 18, n. 9, p. 1314, doi. 10.1038/nm0912-1314
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- Publication type:
- Article
Muscling in: Gene therapies for muscular dystrophy target RNA.
- Published in:
- Nature Medicine, 2010, v. 16, n. 2, p. 170, doi. 10.1038/nm0210-170
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- Publication type:
- Article
News in brief.
- Published in:
- Nature Medicine, 2008, v. 14, n. 10, p. 998, doi. 10.1038/nm1008-998
- Publication type:
- Article
PlGF–MMP-9–expressing cells restore microcirculation and efficacy of cell therapy in aged dystrophic muscle.
- Published in:
- Nature Medicine, 2008, v. 14, n. 9, p. 973, doi. 10.1038/nm.1852
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- Publication type:
- Article
Serotonin and Histamine Therapy Increases Tetanic Forces of Myoblasts, Reduces Muscle Injury, and Improves Grip Strength Performance of Dmd<sup>mdx</sup> Mice.
- Published in:
- Dose-Response, 2015, v. 13, n. 4, p. 1, doi. 10.1177/1559325815616351
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- Publication type:
- Article
Relevant aspects of golden retriever muscular dystrophy for the study of Duchenne muscular dystrophy in humans.
- Published in:
- Ciência Rural, 2017, v. 47, n. 10, p. 1, doi. 10.1590/0103-8478cr20160470
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- Publication type:
- Article
UtroUp is a novel six zinc finger artificial transcription factor that recognises 18 base pairs of the utrophin promoter and efficiently drives utrophin upregulation.
- Published in:
- BMC Molecular Biology, 2013, v. 14, n. 1, p. 1, doi. 10.1186/1471-2199-14-3
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- Publication type:
- Article
Effect of Yoga and Ayurveda on Duchenne Muscular Dystrophy.
- Published in:
- 2011
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- Publication type:
- Letter
Development of Antisense-Mediated Exon Skipping as a Treatment for Duchenne Muscular Dystrophy.
- Published in:
- Annals of the New York Academy of Sciences, 2009, v. 1175, n. 1, p. 71, doi. 10.1111/j.1749-6632.2009.04973.x
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- Publication type:
- Article
Peptide-Morpholino Conjugate: A Promising Therapeutic for Duchenne Muscular Dystrophy.
- Published in:
- Annals of the New York Academy of Sciences, 2009, v. 1175, n. 1, p. 55, doi. 10.1111/j.1749-6632.2009.04976.x
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- Publication type:
- Article
Elemental analysis of biological tissues of Dmd/J and C57BL/6J mice strains investigated by neutron activation analysis.
- Published in:
- Journal of Radioanalytical & Nuclear Chemistry, 2013, v. 296, n. 1, p. 579, doi. 10.1007/s10967-012-2005-6
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- Publication type:
- Article
Analysis of fatty infiltration and inflammation of the pelvic and thigh muscles in boys with Duchenne muscular dystrophy (DMD): grading of disease involvement on MR imaging and correlation with clinical assessments.
- Published in:
- Pediatric Radiology, 2013, v. 43, n. 10, p. 1327, doi. 10.1007/s00247-013-2696-z
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- Publication type:
- Article
Intraarterial Injection of Muscle-derived CD34[sup +]Sca-1[sup +] Stem Cells Restores Dystrophin....
- Published in:
- Journal of Cell Biology, 2001, v. 152, n. 2, p. 335, doi. 10.1083/jcb.152.2.335
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- Publication type:
- Article
Assisted Bicycle Training Delays Functional Deterioration in Boys With Duchenne Muscular Dystrophy: The Randomized Controlled Trial “No Use Is Disuse”.
- Published in:
- Neurorehabilitation & Neural Repair, 2013, v. 27, n. 9, p. 816, doi. 10.1177/1545968313496326
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- Publication type:
- Article
Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics.
- Published in:
- Scientific Reports, 2015, p. 17014, doi. 10.1038/srep17014
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- Publication type:
- Article
Beneficial cilostazol therapeutic effects in mdx dystrophic skeletal muscle.
- Published in:
- Clinical & Experimental Pharmacology & Physiology, 2016, v. 43, n. 2, p. 259, doi. 10.1111/1440-1681.12521
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- Publication type:
- Article